Med-Chemist

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

2026-08-05T10:17:10.970+05:30

Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced the submission to the U.S. Food and Drug Administration (FDA) of the Company's NDA for neladalkib, an investigational ALK-selective inhibitor, in TKI pre-treated advanced ALK-positive NSCLC.

New Drug Application (NDA) based on data in TKI pre-treated patients from the global ALKOVE-1 Phase 1/2 clinical trial

"The advancement of neladalkib from first clinical trial initiation to NDA submission in less than four years represents a remarkable pace in oncology drug development, underscoring the vigor and urgency our team brought to this program and our deep commitment to the ALK-positive NSCLC community," said Darlene Noci, A.L.M., Chief Development Officer at Nuvalent. "We would like to extend our sincere gratitude to the patients, families and investigators who have made this progress possible, and are committed to working closely with the FDA throughout the NDA review process toward our goal of bringing neladalkib to patients as quickly as possible."

The application is based on data in TKI pre-treated patients with advanced ALK-positive NSCLC treated with neladalkib in the global, registration-directed ALKOVE-1 Phase 1/2 clinical trial. In this population, neladalkib demonstrated encouraging overall activity, including intracranial responses, the ability to address key drivers of disease progression, and a generally well-tolerated safety profile consistent with its ALK-selective, TRK-sparing design. The company plans to share detailed results at a future medical meeting.

Neladalkib has received breakthrough therapy designation from the FDA for the treatment of patients with locally advanced or metastatic ALK-positive NSCLC who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

About Neladalkib

Neladalkib is an investigational, brain-penetrant, ALK-selective inhibitor created with the aim to overcome limitations observed with currently available ALK inhibitors. Neladalkib is designed to remain active in tumors that have developed resistance to first-, second-, and third-generation ALK inhibitors, including tumors with single or compound treatment-emergent ALK mutations such as G1202R. In addition, neladalkib is designed for central nervous system (CNS) penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ALK inhibitors and to drive deep, durable responses for patients across all lines of therapy. Neladalkib has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

https://go.drugbank.com/drugs/DB16688

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

Aldeyra Therapeutics Receives Complete Response Letter from the U.S. Food and Drug Administration for the Reproxalap New Drug Application for the Treatment of Signs and Symptoms of Dry Eye Disease

2026-08-03T12:02:31.740+05:30

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to discovering and developing innovative therapies designed to treat immune-mediated diseases, announced receipt of a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) of reproxalap, an investigational drug candidate, for the treatment of dry eye disease. The CRL stated that there is “a lack of substantial evidence consisting of adequate and well-controlled investigations … that the drug product will have the effect it purports or is represented to have under the conditions of use prescribed, recommended, or suggested in its proposed labeling” and that “the application has failed to demonstrate efficacy in adequate and well controlled studies in the treatment of signs and symptoms of dry eye disease.” The letter also stated that the “inconsistency of study results raises serious concerns about the reliability and meaningfulness of the positive findings” and that the “totality of evidence from the completed clinical trials does not support the effectiveness of the product.” Consistent with prior NDA reviews of reproxalap, no safety or manufacturing concerns were identified.

During the NDA review, label drafts were provided by the FDA in December 2025 and again in March 2026. Aldeyra does not believe that label negotiations were completed.

The FDA recommended that the reasons for failure in certain trials be explored, and that populations or certain conditions in which reproxalap may be effective be identified. The FDA did not recommend conducting additional trials or request submission of additional confirmatory evidence. As such, Aldeyra does not currently expect to pursue additional clinical trials, and intends to expeditiously request a Type A meeting to understand the actions needed for NDA approval. Per Prescription Drug User Fee Act (PDUFA) goals, the target Type A meeting date is within 30 days of receipt of the meeting request.

“To the thousands of American and Canadian patients who participated in our clinical trials and to the tens of millions of patients with dry eye disease worldwide, I want to assure you that we will work with urgency to support the FDA in enabling market access to what is, to our knowledge, the only drug with clinical activity within minutes of administration in patients with dry eye disease, a condition that is today treated with medications that require weeks or months of treatment to achieve even modest improvement,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra.

https://go.drugbank.com/drugs/DB16688

Aldeyra Therapeutics Receives Complete Response Letter from the U.S. Food and Drug Administration for the Reproxalap New Drug Application for the Treatment of Signs and Symptoms of Dry Eye Disease

Cogent Biosciences Announces Submission of New Drug Application for Bezuclastinib in Gastrointestinal Stromal Tumors (GIST)

2026-08-01T14:56:33.889+05:30

Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, announced the completion of the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in patients with Gastrointestinal Stromal Tumors (GIST) who have received prior treatment with imatinib. Based on the positive results from the PEAK trial, the bezuclastinib NDA was submitted under the FDA’s Real-Time Oncology Review (RTOR) program, which is intended to enable a more streamlined review process. Bezuclastinib was also granted Breakthrough Therapy Designation as a treatment for GIST earlier this year.

Bezuclastinib NDA submitted under the FDA’s RTOR program based on positive results from Phase 3 PEAK trial; bezuclastinib previously granted Breakthrough Therapy Designation in GIST

Bezuclastinib combination demonstrated 16.5 month mPFS and 46% ORR in imatinib-resistant GIST patients, dramatically improving upon the current standard of care

“We are excited to complete our PEAK NDA submission which marks a significant step toward bringing a new therapy to patients with second-line GIST,” said Andrew Robbins, President and Chief Executive Officer. “Based on the strength of the PEAK data, we believe the bezuclastinib combination has the potential to meaningfully change the treatment landscape for these patients. We are grateful to the patients, investigators, and study teams who made this possible.”

Pivotal data from PEAK, a global, randomized Phase 3 clinical trial evaluating bezuclastinib in combination with sunitinib vs. sunitinib monotherapy in patients with GIST who have received prior treatment with imatinib, were reported in November 2025. As disclosed in the top-line results, the bezuclastinib combination demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression free survival (PFS), reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39 – 0.65). mPFS, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy. Additionally, the bezuclastinib combination demonstrated an unprecedented ORR in imatinib-resistant patients, with 46% of patients treated with the bezuclastinib combination achieving an objective response compared to 26% of patients treated with sunitinib. The bezuclastinib combination was generally well tolerated, and no unique risks were observed with the novel combination when compared to the known safety profile of sunitinib. Data for overall survival remains immature.

At the time of data cutoff, based on the number of ongoing patients receiving treatment on the bezuclastinib combination arm, the estimated mean duration of treatment for the bezuclastinib combination is projected to exceed 19 months.

Cogent plans to present full results from the PEAK trial at a major medical meeting during the first half of 2026. Additionally, Cogent is on track this quarter to initiate a Phase 2 trial investigating the benefit of the bezuclastinib plus sunitinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib. The NDA submission with bezuclastinib in Advanced Systemic Mastocytosis (AdvSM) also remains on track for the first half of 2026.

Bezuclastinib - Expanded Access Program

Working with the FDA, Cogent has established active Expanded Access Programs (EAPs) for U.S. patients with GIST or SM who meet disease-specific criteria and could benefit from treatment with bezuclastinib or the combination of bezuclastinib and sunitinib. A growing number of sites now offer access to the bezuclastinib EAPs. For more information please visit: https://www.cogentbio.com/bezuclastinib-program-development/#our-expanded-access-policy

Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Cogent also announced today that, on March 30, 2026, the Compensation Committee of Cogent’s Board of Directors, made up entirely of independent directors, approved the grants of “inducement” equity awards to six new employees under the company’s 2020 Inducement Plan with a grant date of March 30, 2026. The awards were approved in accordance with Listing Rule 5635(c)(4) of the corporate governance rules of the Nasdaq Stock Market. The employees received, in the aggregate, (i) nonqualified options to purchase 21,100 shares of Cogent common stock and (ii) 15,700 restricted stock units (RSUs). Each option has a 10-year term, an exercise price equal to the closing price of Cogent’s common stock on the grant date, and a 4-year vesting schedule with 25% vesting on the 1-year anniversary of the grant date and the remainder vesting in equal monthly installments over the subsequent 36 months, provided such employee remains employed through each such vesting date. The RSUs vest annually in equal installments over 4 years from the grant date, provided such employee remains employed through each such vesting date.

https://en.wikipedia.org/wiki/Bezuclastinib

Cogent Biosciences Announces Submission of New Drug Application for Bezuclastinib in Gastrointestinal Stromal Tumors (GIST)

Nanomedicine offers targeted solutions for breast cancer treatment

2026-07-30T10:16:45.118+05:30

Breast cancer (BCA) is one of the most common cancers worldwide, with high mortality and morbidity in women. This review focuses on the applications of nanotechnology, nanomaterials (NMs), and nanoparticles (NPs) in BCA diagnosis and therapy. Nanotechnologies, nanocarriers, and nano-encapsulation versus conventional counterparts are discussed. Various drug formulations into lipid NPs, nanoemulsions, polymeric NPs, and metal-based NPs enhance bioavailability and therapeutic efficacy, overcoming limitations of conventional formulations. Clinical specialists have achieved improved outcomes in BCA detection and monitoring using nanotechnology, ultimately improving patients' quality of life.

Introduction

Cancer is a leading cause of death globally. Breast cancer accounts for 30% of all cancer cases and 15% of cancer‑related deaths in women. The PI3K/AKT/mTOR signaling pathway plays a crucial role in BCA development and progression. Nanomedicine applies NMs and NPs for prevention, diagnosis, and treatment. This review addresses challenges of conventional therapies (lack of target specificity, drug resistance, systemic toxicity) and highlights how nanotechnology overcomes these limitations.

General Aspects of NMs and NPs

NMs have at least one dimension in 1–100 nm and a large surface‑to‑volume ratio, conferring novel properties. Reducing particle size increases solubility and surface interactions. Nanotechnology improves pharmacokinetics, enables targeted delivery, enhances permeability and retention effects in tumors, and reduces required drug doses.

Different Aspects of BCA

Statistics: GLOBOCAN 2022 reported 2,296,840 new BCA cases (ASIR 46.8 per 10⁵) and 666,103 deaths (ASMR 12.7 per 10⁵) worldwide.
Molecular Subtypes: BCA is classified by hormone receptor and HER2 status: Luminal A (~40%, ER⁺/PR⁺, HER2⁻), Luminal B (~20%, ER⁺/PR⁺, HER2⁺/⁻), HER2‑enriched (~10‑15%, ER⁻/PR⁻, HER2⁺), and triple‑negative breast cancer (TNBCA, ~15‑20%, ER⁻/PR⁻/HER2⁻). TNBCA is aggressive, occurs in younger women, has high recurrence and metastasis rates, and lacks targetable proteins.
Challenges: Treatment resistance, recurrence, adverse effects, low cellular absorption, and multidrug resistance.
Therapies: Surgery, chemotherapy, radiotherapy, hormonal therapy, and immunotherapy. Nanotechnology offers innovative carriers to overcome limitations.

Carriers and Nanocarriers for Drug Delivery

Conventional carriers have limited tumor response and affect normal cells. Nanocarriers include lipid nanoparticles (LNPs), nanoemulsions (NEs), polymeric NMs, and metallic NPs. They enhance drug stability, absorption, encapsulation efficiency, bioavailability, and controlled release. NEs improve oral delivery of poorly soluble drugs and reduce toxicity.

Nanocarriers for BCA

Chitosan‑based nanocarriers exploit electrostatic interactions with cancer cells, enhance cellular uptake, and open tight junctions. Quaternary ammonium chitosan improves penetration. Chitosan NPs deliver genes, drugs, and natural compounds; induce phototherapy‑mediated tumor ablation; and support combination therapy.

Clinical results: Nanocarriers improved drug delivery and outcomes. Photothermal nanomaterials (PTT) with nanotechnology enhanced metastatic BCA treatment, reduced damage to healthy cells, and synergized with chemotherapy/immunotherapy. Cyclophosphamide NEs in rats showed remarkable tumor reduction. Exemestane‑loaded polymer‑lipid hybrid nanoparticles (PLH NPs) improved oral bioavailability (>3.5‑fold) and tumor inhibition (62% vs. 31% for conventional suspension) in mice.

Major Metallic Nanocarriers

Gold (Au) NPs: Biocompatible, easy surface modification, effective against TNBCA via Rad6 conjugation inducing mitochondrial dysfunction. Clinical translation limited by toxicity in liver, kidneys, spleen.
Silver (Ag) NPs: High photon attenuation; ethyl cellulose‑coated Ag NPs inhibited TNF‑α in BCA cells.
Copper (Cu) NPs: Bioactive; 5‑fluorouracil loaded into β‑cyclodextrin‑Cu NPs showed sustained release and anticancer activity against TNBCA.
Iron oxide (Fe₃O₄) NPs: Magnetic core‑shell NPs (Fe₃O₄‑poly(N‑isopropylacrylamide)‑grafted chitosan) delivered methotrexate with 94% entrapment efficiency; enhanced antitumor activity against MCF‑7 cells at 40°C and pH 5.5.

REF: https://www.xiahepublishing.com/2996-3427/OnA-2025-00027

Nanomedicine offers targeted solutions for breast cancer treatment

Metformin mimics exercise-related metabolic effects in prostate cancer patients

2026-07-29T13:20:23.025+05:30

In continuation of my update on metformin

A new study has found that metformin, a widely prescribed diabetes drug, may mimic one of exercise's core biological effects in men with prostate cancer, raising levels of a molecule tied to energy balance and weight control even when patients are inactive. The findings suggest metformin could help counter the metabolic strain of hormone therapy, when fatigue and other side effects often limit physical activity.

Led by physician-scientists at Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, the study appears in the journal EMBO Molecular Medicine.

Exercise is one of the most reliable ways to support health during cancer treatment. It helps regulate weight, blood sugar and cardiovascular health-factors that shape how patients feel during therapy and how well they recover afterward.

For many people with cancer, however, regular exercise isn't always feasible. Fatigue, hormone therapy, pain or advanced disease can limit physical activity precisely when metabolic health becomes most important.

That reality has led researchers to ask a practical question: if exercise confers its benefits through specific biological signals, could some of those signals be activated in other ways?

According to the research, the answer may be yes. Sylvester investigators report that metformin raises levels of a naturally occurring molecule involved in how the body manages energy and weight in prostate cancer patients.

The finding does not suggest that a pill can replace physical activity. Instead, it offers insight into the internal pathways that underlie exercise's metabolic benefits-and how those pathways might still be engaged when movement is limited.

This study reflects what's possible when laboratory science, metabolic biology and clinical investigation are intentionally brought together for transdisciplinary studies. By working across Sylvester's Tumor Biology, Cancer Epigenetics and Translational & Clinical Oncology programs, we were able to link a well-defined molecular signal to real patient data. The result isn't a new cancer biomarker, but a clearer understanding of how a widely used drug may support metabolic health during prostate cancer treatment-an outcome that matters to patients and clinicians alike."
Marijo Bilusic, M.D., Ph.D., Sylvester researcher and first author, genitourinary medical oncologist and professor of medicine and medical oncology at the Miller School.

At the center of the collaborative, team-science study is a molecule called N-lactoyl-phenylalanine, or Lac-Phe. While its name is technical, its role is relatively simple.

Lac‑Phe is produced when the body is under metabolic demand. It forms when lactate-a substance that accumulates during exertion-combines with phenylalanine, a basic building block of protein. Scientists first took notice of Lac‑Phe because its levels spike after intense exercise, coinciding with shifts in energy use and appetite regulation.

https://en.wikipedia.org/wiki/Metformin

Metformin mimics exercise-related metabolic effects in prostate cancer patients

FDA Approves Komzifti (ziftomenib) for the Treatment of Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

2026-07-28T16:03:53.806+05:30

Kura Oncology, Inc. (Nasdaq: KURA) and Kyowa Kirin Co., Ltd. (TSE: 4151) announced the U.S. Food and Drug Administration (FDA) approval of Komzifti™ (ziftomenib) for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options. Komzifti is the first and only once-daily, oral menin inhibitor approved for R/R NPM1-mutated (NPM1-m) AML, a devastating blood cancer with limited treatment options.

“Komzifti combines compelling efficacy, a favorable safety profile, compatibility with concomitant medications, and convenient once-daily oral administration in a population with few effective treatment options. These features highlight Komzifti’s potential to serve as the menin inhibitor of choice in its approved indication,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “Together with our partner, Kyowa Kirin, we remain committed to advancing development of Komzifti across the treatment continuum for AML, where its best-in-class profile offers potential for even greater impact in combination regimens and earlier lines of therapy. We are fully prepared to launch Komzifti today and deliver this new medicine to patients in need.”

NPM1 mutations are among the most common founder mutations in AML, occurring in approximately 30% of cases. Historically, approximately 20% of patients with NPM1-m AML do not respond to front-line therapy. Of those who do respond, 70% will relapse within 3 years, most within 12 months. Early relapse and declining survival with each recurrence underscore the urgent need for treatment approaches that deliver lasting remission.

Approval is supported by the pivotal KOMET-001 trial (NCT04067336), which evaluated Komzifti’s safety and efficacy in 112 R/R NPM1-m AML patients. The rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) was 21.4% (95% CI: 14.2, 30.2). The median duration of CR+CRh was 5.0 months (95% CI: 1.9, 8.1) and the median time to first response in patients who achieved a CR or CRh was 2.7 months (range: 0.9 to 15 months). 88% of patients who achieved CR or CRh did so within 6 months of initiating Komzifti. These data from the Prescribing Information are generally consistent with findings recently published in the Journal of Clinical Oncology.1

“Komzifti addresses a critical need for adult patients with R/R NPM1-m AML, many of whom are older and unable to tolerate intensive chemotherapy or transplant,” said Eunice Wang, M.D., Chief of the Leukemia Service and Professor of Oncology at Roswell Park Comprehensive Cancer Center. “The clinical data demonstrate deep and durable responses with a manageable safety profile, including no drug-drug interactions and no Boxed Warnings for QTc prolongation or Torsades de Pointes – key advantages for patients on multiple concurrent medications. This approval equips physicians with a new oral therapy to integrate into care and improve outcomes for this vulnerable patient population.”

The most common adverse reactions (≥20%), including laboratory abnormalities, were aspartate aminotransferase increased, infection without an identified pathogen, potassium decreased, albumin decreased, alanine aminotransferase increased, sodium decreased, creatinine increased, alkaline phosphatase increased, hemorrhage, diarrhea, nausea, fatigue, edema, bacterial infection, musculoskeletal pain, bilirubin increased, potassium increased, differentiation syndrome, pruritus, febrile neutropenia, and transaminases increased. Komzifti includes a Boxed Warning for differentiation syndrome, a well-studied mechanism-based risk in drugs that restore differentiation. Absence of clinically meaningful drug-drug interactions can ease the use of Komzifti with concomitant therapies, including those that cause QTc interval prolongation. QTc interval prolongation was ≤ Grade 3 in 12% of patients and no Grade 4 or Grade 5 QTc interval prolongation was reported. QTc interval prolongation of any cause occurred in 10% of the 70 patients 65 years of age or older.

“The approval of Komzifti underscores our commitment to advancing precision medicines to address the genetic drivers of disease in hematology and oncology,” said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. “In AML, where many patients face severe disease progression and limited treatment options, the evolution toward targeted therapies such as Komzifti represents a major step forward and offers potential to transform existing standards of care. We are proud to partner with Kura Oncology in bringing this important therapy to patients and their families.”

In November 2024, Kura Oncology and Kyowa Kirin entered into a global strategic collaboration to develop and commercialize Komzifti. The collaboration builds on Kyowa Kirin’s leadership and expertise in hematologic malignancies. Under the terms of the collaboration, Kura leads development, regulatory and commercial strategy in the U.S. and is responsible for manufacturing Komzifti. The companies will jointly perform certain commercialization activities in accordance with a co-created U.S. territory commercialization plan. Outside the U.S., Kyowa Kirin leads development, regulatory and commercial strategy and is responsible for commercializing Komzifti.

As part of its commitment to helping patients access Komzifti, Kura has established a support program, Kura RxKonnect™, to minimize barriers to access and reimbursement for appropriate patients prescribed Komzifti. Kura RxKonnect is available 8:00 am – 8:00 pm ET by phone at (844) KuraPSP (844-587-2777) or online at www.KuraRxKonnect.com.

https://en.wikipedia.org/wiki/Ziftomenib

FDA Approves Komzifti (ziftomenib) for the Treatment of Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

FDA Grants Accelerated Approval to Hyrnuo (sevabertinib) for Non-Squamous Non-Small Cell Lung Cancer

2026-07-25T09:56:21.019+05:30

Following Priority Review and Breakthrough Therapy Designation, the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Hyrnuo (sevabertinib), for the treatment of adult patients with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have human epidermal growth factor receptor 2 (HER2) tyrosine kinase domain activating mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy. Sevabertinib is an oral, reversible, small molecule, tyrosine kinase inhibitor (TKI).

“The FDA’s approval of sevabertinib elevates the standard of care and provides a new treatment option for patients living with HER2-mutant NSCLC, which is a challenging disease with limited therapies available,” said SOHO-01 lead trialist, Xiuning Le, MD, PhD, The University of Texas MD Anderson Cancer Center, Houston, Texas. “Clinical results from the SOHO-01 trial showed that sevabertinib is effective with a manageable safety profile.”

The FDA approved this indication for sevabertinib under accelerated approval based on objective response rate (ORR) and duration of response (DOR) data from the ongoing Phase I/II SOHO-01 trial (NCT05099172) in patients with advanced NSCLC harboring a HER2-activating mutation, with disease progression after ≥1 systemic therapies for advanced disease. Continued approval of sevabertinib for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Results from the Phase I/II SOHO-01 trial demonstrated for patients naïve to HER2 targeted therapy an ORR of 71% (95% CI: 59%-82%, N=70); 2.9% of patients had a complete response and 69% of patients had a partial response. Median DOR was 9.2 months (95% CI, 6.3–15.0, N=50). Sevabertinib demonstrated a manageable safety profile with a 3.7% discontinuation rate. In the pooled safety population, the most common (> 20%) adverse reactions were diarrhea (87%), rash (66%), paronychia (33%), stomatitis (29%) and nausea (21%).

Positive results from the Phase I/II SOHO-01 trial were presented at the European Society for Medical Oncology (ESMO) Congress 2025 and simultaneously published in the New England Journal of Medicine.0F[1]

“Globally, it is estimated that up to 84,000 people are diagnosed with NSCLC harboring a HER2 mutation each year,” said Christian Rommel, Ph.D., Head of Research and Development at Bayer’s Pharmaceuticals Division. “At Bayer our oncology R&D strategy is centered on precision drug development which is designed to help us address critical unmet medical needs through the advancement of innovative therapies. Today's FDA approval of sevabertinib exemplifies this approach, delivering a targeted treatment specifically for patients with advanced HER2-mutant NSCLC. This significant milestone underscores our unwavering commitment to transforming cancer care by delivering meaningful therapies that have the potential to improve outcomes and extend survival for people living with cancer.”

In 2024, both the U.S. FDA and the CDE in China granted sevabertinib Breakthrough Therapy designation indicating that the compound has the potential to provide substantial improvement over existing therapies in NSCLC with HER2-activating mutations. China accepted a new drug application (NDA) for sevabertinib in July 2025.

https://en.wikipedia.org/wiki/Sevabertinib

FDA Grants Accelerated Approval to Hyrnuo (sevabertinib) for Non-Squamous Non-Small Cell Lung Cancer

FDA Approves Cardamyst (etripamil) Nasal Spray for Adults with Paroxysmal Supraventricular Tachycardia

2026-07-24T09:46:47.201+05:30

Milestone Pharmaceuticals Inc. (Nasdaq: MIST) announced the U.S. Food and Drug Administration (FDA) approved its first commercial product, Cardamyst (etripamil) nasal spray, a prescription medication for the conversion of acute symptomatic episodes of paroxysmal supraventricular tachycardia (PSVT) to sinus rhythm in adults. This approval marks the first time that more than two million Americans with PSVT will have a rapid-acting treatment option they can self-administer outside the emergency department or other healthcare setting. Cardamyst is expected to be available in retail pharmacies in the first quarter of 2026.

Cardamyst nasal spray is a novel and rapid-acting calcium channel blocker delivered when needed to treat often highly symptomatic and unpredictable episodes of PSVT. With Cardamyst, adults with PSVT can be prepared wherever and whenever episodes occur, providing them with active management and a greater sense of control of their condition.

“Cardamyst is a novel at-the-ready treatment option that addresses the unpredictable impact of PSVT by offering patients the freedom to manage episodes anytime and anywhere,” said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. “The FDA approval of Cardamyst is a watershed moment for Milestone and a gratifying event for our team members, patients, clinical investigators, and health care providers who participated in the development program, all of whom I sincerely thank for their dedication, counsel, and collaboration toward this important achievement.”

“Some people with PSVT have endured years of anxiety, fearing their next episode and the stress and disruption of emergency department visits,” said James Ip, M.D., FACC, FHRS, an etripamil investigator. “Cardamyst will give many of them the ability to administer a medication themselves that can quickly stop their PSVT episode and potentially avoid a hospital trip or a call to emergency services.”

Cardamyst Clinical Data

The FDA approval of Cardamyst is supported by a robust clinical trial program based on safety data from more than 1,800 participants and more than 2,000 episodes of PSVT. This includes the successful Phase 3 RAPID trial, a global, randomized, double-blind comparison of Cardamyst vs. placebo, published in The Lancet in 2023. In clinical studies, participants using Cardamyst were two times more likely to convert symptomatic PSVT to sinus rhythm and did so more than three times faster compared with placebo. The RAPID trial achieved its primary endpoint with 64% of those who self-administered Cardamyst (N=99) converting from supraventricular tachycardia (SVT) to sinus rhythm within 30 minutes compared to 31% on placebo (N=85) (HR = 2.62; p<0.001). At one hour, the benefit was demonstrated in 73% of participants. In addition, significant reductions in time to conversion in those who took Cardamyst were evident early and durable, with a median time to conversion of 17 minutes (95% CI: 13.4, 26.5) for those treated with Cardamyst vs. 54 minutes (95% CI: 38.7, 87.3) for those treated with placebo.

A consistent safety profile and treatment effects were observed across all subgroups, including participants concurrently on beta blockers or calcium channel blockers. The most frequent adverse events occurring in ≥5% of participants in randomized clinical trials were mild-to-moderate and transient in nature, including local-site nasal discomfort, nasal congestion, rhinorrhea, throat irritation, and epistaxis. Less than 2% of trial participants discontinued therapy due to adverse events.

For more safety information about Cardamyst, please see Important Safety Information below. For the latest information about the product availability, please see https://milestonepharma.com/.

PSVT, also called SVT, is characterized by episodes of abnormally fast heart rate. Most people with PSVT experience multiple sustained episodes that require treatment on an annual basis. Until now, successful treatment options typically required IV administration in healthcare settings, creating stress and costs for patients and their insurers. Cardamyst offers a new approach to treat episodes of PSVT, enabling adults to self-administer the medication at the onset of symptoms. For more information, please visit SVTHearttoHeart.com.

“Our goal is that Cardamyst will become a trusted and essential solution for healthcare providers and their patients,” said Lorenz Muller, Chief Commercial Officer of Milestone. “Our team is focused on making Cardamyst available to adults with PSVT as quickly as possible, including actively working to secure insurance coverage and begin the distribution of the product through retail pharmacies.”

Annette Greene, Cardamyst clinical trial participant and administrator of the Supraventricular Tachycardia Group on Facebook, with more than 30,000 members, said, “Adults with SVT have been waiting a long time for the day when they can confidently self-administer Cardamyst to treat their SVT episodes. It is very exciting that the day has become a reality.”

Regarding future R&D, Milestone is poised to enter a Phase 3 program in atrial fibrillation with rapid ventricular rate (AFib-RVR), particularly on the strength of the successful ReVeRA Phase 2 trial in AFib-RVR, results of which were published in Circulation: Arrhythmia and Electrophysiology. Incorporating FDA’s guidance, Milestone has developed a Phase 3 registrational program to evaluate self-administered etripamil as a potential treatment for patients with AFib-RVR. When completed, the Company will be following a supplemental New Drug Application (sNDA) regulatory approval pathway for a potential second indication for etripamil in AFib-RVR. As such, Milestone will leverage the approved PSVT indication and the PSVT program data, along with a single pivotal Phase 3 study in patients with AFib-RVR.

https://en.wikipedia.org/wiki/Etripamil

FDA Approves Cardamyst (etripamil) Nasal Spray for Adults with Paroxysmal Supraventricular Tachycardia

FDA Approves Nuzolvence (zoliflodacin) for the Treatment of Uncomplicated Urogenital Gonorrhea in Adults and Adolescents

2026-07-23T10:35:56.561+05:30

Innoviva Specialty Therapeutics, a subsidiary of Innoviva, Inc. (NASDAQ: INVA), announced the U.S. Food and Drug Administration (FDA) approval of Nuzolvence (zoliflodacin) for oral suspension, a first-in-class, single-dose oral medication for the treatment of uncomplicated urogenital gonorrhea in adults and pediatric patients 12 years and older weighing at least 35 kg. The development of Nuzolvence was part of a private, not-for-profit collaboration with The Global Antibiotic Research and Development Partnership (GARDP), which sponsored and led the Phase 3 clinical trial that supported FDA approval.

“The FDA’s approval of Nuzolvence marks a pivotal moment for patients and the broader healthcare community managing gonorrhea infections. For the first time in decades, both patients and their healthcare providers will have a single-dose, oral treatment option for uncomplicated urogenital gonorrhea,” said David Altarac, M.D., Chief Medical Officer, Innoviva Specialty Therapeutics. “This achievement underscores our commitment to advancing innovative therapies for infectious diseases and fighting antimicrobial resistance. It highlights the strength of our development capabilities, as well as our collaboration with GARDP and the global scientific community.”

Gonorrhea is the second most common sexually transmitted bacterial infection worldwide, with more than 82 million new cases each year. In the United States alone, the Centers for Disease Control and Prevention (CDC) estimates that over 543,000 cases are reported each year and over 1 million incident cases occur annually,3 underscoring the significant public health impact. The highest rates are seen among sexually active men ages 20-24, though anyone who has unprotected sex can be at risk.4 Without timely treatment, gonorrhea can lead to serious and potentially permanent health complications.

"The decades-long absence of new gonorrhea treatments, combined with rising global antibiotic resistance, has created significant challenges in managing this common but potentially serious sexually transmitted infection," said Edward W. Hook III, M.D., Professor Emeritus of Medicine, University of Alabama at Birmingham, and lead investigator of the Phase 3 Nuzolvence trial. “In the pivotal Phase 3 study, Nuzolvence demonstrated non-inferiority compared to the current standard injectable therapy, including in infections caused by drug-resistant strains, while offering the convenience of a single oral dose.”

“A new antibiotic that does not require injection and can be used for patients who are allergic to penicillin or related drugs meets two important unmet needs in the treatment of gonorrhea,” added Dr. Hook.

The emergence and spread of global drug-resistant infections have led the World Health Organization (WHO) to identify antimicrobial resistance as one of the 10 most critical global health threats.5 The bacterium Neisseria gonorrhoeae has developed resistance to most classes of antibiotics used to treat these infections, including cephalosporins such as ceftriaxone, an injectable treatment, which is the currently recommended first line therapy for uncomplicated urogenital gonorrhea.

The Company plans to commercialize Nuzolvence in the second half of 2026, either in collaboration with a commercialization partner or independently.

https://en.wikipedia.org/wiki/Zoliflodacin

FDA Approves Nuzolvence (zoliflodacin) for the Treatment of Uncomplicated Urogenital Gonorrhea in Adults and Adolescents

FDA Approves Myqorzo (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy

2026-07-22T09:47:49.842+05:30

Cytokinetics, Incorporated (Nasdaq: CYTK) announced the U.S. Food and Drug Administration (FDA) approval of Myqorzo™ (aficamten, 5 mg, 10 mg, 15 mg, 20 mg tablets for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. Myqorzo is an allosteric and reversible inhibitor of cardiac myosin motor activity. In patients with oHCM, myosin inhibition with Myqorzo reduces cardiac contractility and left ventricular outflow tract (LVOT) obstruction.

“This is a historic moment for our company and for the patients we serve, as we fulfill our promise to translate our science into medicines that may make a meaningful difference in patients’ lives,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “Our first FDA approval stands as a testament to the strength of our science and the bold, trailblazing research that has defined Cytokinetics’ leadership in muscle biology. I’m pleased that the approved label and REMS reflect the distinct characteristics of Myqorzo including a straightforward, flexible dosing regimen, no requirement for drug-drug interaction monitoring and a predictable safety profile. I am profoundly grateful for the many years of passion and persistence shown by patients with obstructive HCM, as well as healthcare professionals, advocates, partners and employees who have contributed so importantly to reaching this key milestone.”

The full U.S. Prescribing Information for Myqorzo includes a Boxed WARNING for the risk of heart failure. Myqorzo reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction. Echocardiogram assessments are required prior to and during treatment with Myqorzo to monitor for systolic dysfunction. Initiation of Myqorzo in patients LVEF <55% is not recommended. Decrease the dose of Myqorzo if LVEF <50% and ≥40%. Interrupt the dose of Myqorzo if LVEF <40% or if the patient experiences heart failure symptoms or worsening clinical status due to systolic dysfunction. Because of the risk of heart failure due to systolic dysfunction, Myqorzo is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Myqorzo REMS Program. Please see additional Important Safety Information including Boxed WARNING below.

Myqorzo is expected to be available in the U.S. in the second half of January 2026. Cytokinetics will support patients with Myqorzo & You™, a personalized program for patients prescribed Myqorzo in the U.S. to help navigate the treatment journey, provide disease and product education, and offer support with insurance benefits investigations or financial assistance for those eligible. For more information, call 833-Myqorzo (833-697-6796).

“HCM is a heart muscle disease associated with a significant symptom burden. This approval of a new drug, Myqorzo, represents a meaningful addition to the treatment options available for symptomatic obstructive HCM patients,” said Martin Maron, M.D., Director, Hypertrophic Cardiomyopathy Center, Lahey Hospital and Medical Center, and Principal Investigator of SEQUOIA-HCM. “In SEQUOIA-HCM, Myqorzo improved exercise capacity and reduced symptoms while also being well-tolerated. For these reasons, Myqorzo represents an important step forward in how we care for people living with obstructive HCM.”

https://en.wikipedia.org/wiki/Aficamten

FDA Approves Myqorzo (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy

FDA Approves Aqvesme (mitapivat) for the Treatment of Anemia in Adults with Alpha- or Beta-Thalassemia

2026-07-21T09:48:24.413+05:30

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, announced the U.S. Food and Drug Administration (FDA) approval of Aqvesme (mitapivat), an oral pyruvate kinase (PK) activator, for the treatment of anemia in adults with alpha- or beta-thalassemia. With this approved indication, Aqvesme becomes the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.

“Thalassemia is a debilitating disease that demands lifelong management and vigilant monitoring for many life-threatening complications, such as blood clots, heart disease, and liver disease. Despite its severity, treatments have historically been limited, leaving some patients without any options,” said Hanny Al-Samkari, M.D., Peggy S. Blitz Endowed Chair in Hematology/Oncology at Mass General Brigham Cancer Institute, Associate Professor at Harvard Medical School, and an investigator for the mitapivat thalassemia Phase 3 clinical program. “The ENERGIZE and ENERGIZE-T Phase 3 trial results demonstrate that Aqvesme can help address anemia, fatigue, and the need for regular transfusions – key challenges of the disease. Today’s FDA approval represents an important step forward for individuals with thalassemia.”

The FDA approval of Aqvesme in thalassemia is based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively. A total of 452 patients who are representative of the real-world thalassemia population were enrolled in the trials. The ENERGIZE and ENERGIZE-T Phase 3 trials met all primary and key secondary efficacy endpoints, demonstrating that Aqvesme improves hemolytic anemia and a key quality-of-life measure compared to placebo, including significant reductions in transfusion burden and significant improvements in hemoglobin and fatigue.

“Today is a landmark moment for the thalassemia community, bringing forward an innovative, disease-modifying oral medicine to address the urgent needs of people living with this devastating rare blood disorder,” said Brian Goff, Chief Executive Officer, Agios. “With this approval, Aqvesme becomes the only medicine indicated for the treatment of anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. We are deeply grateful to the patients, caregivers, healthcare providers, and advocacy partners who participated in and supported our clinical trials and helped ensure that our efforts reflected their priorities. Our focus now is on ensuring a successful launch and delivering meaningful impact for the community.”

“This year marks 100 years since thalassemia was first described in medical literature, and the progress we’ve made as a community is truly extraordinary,” said Ralph Colasanti, National President, Cooley’s Anemia Foundation. “Innovative medicines like Aqvesme help make this progress possible, offering new hope to patients like me by helping to address the complex needs and challenges of this disease. This approval provides an important new treatment option for the thousands of adults living with thalassemia in the U.S., including those with non-transfusion-dependent thalassemia who previously had no approved alternatives.”

In the ENERGIZE and ENERGIZE-T Phase 3 trials, five patients receiving Aqvesme experienced adverse reactions suggestive of hepatocellular injury (HCI), with two of these patients requiring hospitalization. These adverse reactions occurred within the first six months of exposure, and liver tests improved upon discontinuation of Aqvesme. To mitigate the risk of HCI, Aqvesme is available only through the Aqvesme REMS, a Risk Evaluation and Mitigation Strategy (REMS) program approved by the FDA. The Aqvesme REMS program requires liver tests prior to the first Aqvesme dose, every four weeks thereafter for 24 weeks, and then as clinically indicated. It also includes education and certification requirements for patients, prescribing physicians, and pharmacists, which are common components of REMS programs.

Due to the Aqvesme REMS program, mitapivat will be marketed under the brand name Aqvesme in the U.S. for the thalassemia indication. Mitapivat will continue to be marketed as Pyrukynd® (mitapivat) in the U.S. for the PK deficiency indication, which does not require a REMS program. Outside the U.S., mitapivat will continue to be marketed as Pyrukynd for its PK deficiency and thalassemia indications in regions where it is approved, and will retain this name upon approval in regions currently under regulatory review.

Agios expects Aqvesme to be available in the U.S. in late January 2026, following implementation of the Aqvesme REMS program. For more information, visit Aqvesme.com.

About Thalassemia

Thalassemia is a rare, inherited blood disease that affects the production of hemoglobin, the protein in red blood cells responsible for carrying oxygen throughout the body. The disease is categorized into two main types: alpha-thalassemia and beta-thalassemia, depending on which globin chain of the hemoglobin is affected. By disrupting hemoglobin production, thalassemia reduces the number of circulating red blood cells and shortens their lifespan, which leads to anemia, fatigue, and serious complications.

Some individuals with thalassemia require regular transfusions (classified as transfusion-dependent thalassemia), while others only need them intermittently (classified as non-transfusion-dependent thalassemia). All patients with thalassemia experience a significant disease burden, including comorbidities, reduced quality of life, and shortened life expectancy.

In the U.S., approximately 6,000 adult patients are diagnosed with thalassemia.

About ENERGIZE and ENERGIZE-T

ENERGIZE (NCT04770753) and ENERGIZE-T (NCT04770779) are global, double-blind, placebo-controlled Phase 3 trials evaluating the efficacy and safety of mitapivat in adults with alpha- or beta-thalassemia.

The ENERGIZE trial randomized 194 non-transfusion-dependent alpha- or beta-thalassemia patients 2:1 to receive either mitapivat 100 mg twice daily or placebo. The primary endpoint was hemoglobin response, defined as an increase of ≥1.0 g/dL in average hemoglobin concentration from Week 12 through Week 24 compared with baseline. Key secondary endpoints included changes from baseline in average fatigue scores and in average hemoglobin concentration from Week 12 to Week 24. The trial also assessed safety and tolerability.

The ENERGIZE-T trial randomized 258 transfusion-dependent alpha- or beta-thalassemia patients 2:1 to receive either mitapivat 100 mg twice daily or placebo. The primary endpoint was transfusion reduction response, defined as a ≥50% reduction in transfused red blood cell (RBC) units with a reduction of ≥2 units of RBCs transfused in any consecutive 12-week period through Week 48 compared with baseline. Several transfusion reduction measures were included as key secondary endpoints, and achievement of transfusion independence was a secondary endpoint. The trial also assessed safety and tolerability.

For each trial, patients who completed the double-blind phase had the option to transition into a corresponding open-label extension phase, during which all patients receive mitapivat.

https://en.wikipedia.org/wiki/Mitapivat

FDA Approves Aqvesme (mitapivat) for the Treatment of Anemia in Adults with Alpha- or Beta-Thalassemia

Egetis Announces FDA Acceptance and Priority Review of NDA for Emcitate (tiratricol) for MCT8 Deficiency

2026-07-20T09:44:36.878+05:30

Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (NASDAQ Stockholm: EGTX), today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of its New Drug Application (NDA) for Emcitate® (tiratricol) for the treatment of MCT8 deficiency. Egetis completed the rolling NDA in January 2026. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date, or FDA decision date, of September 28, 2026.

NDA submission for Emcitate® successfully validated by the FDA

Priority Review granted and PDUFA target action date set to September 28, 2026

Emcitate® (tiratricol) is eligible to receive a Priority Review Voucher (PRV) upon approval

If approved, Emcitate® (tiratricol) would become the first approved treatment in the United States for MCT8 deficiency

Nicklas Westerholm, CEO of Egetis, commented: “MCT8 deficiency is a rare, devastating, life-shortening disorder with no approved treatment in the U.S. The acceptance of our NDA with Priority Review represents an important step in the path to making Emcitate® (tiratricol) available to MCT8 deficiency patients in the U.S. The Priority Review also highlights the robust dataset we have in this rare genetic disease, with the NDA based on results from Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study, EMC Survival Study and the U.S. Expanded Access Program. We look forward to collaborating with the FDA during the NDA Priority Review process and will in parallel continue to build our medical affairs and market access capabilities towards a potential U.S. launch in the fourth quarter of this year.”

A Priority Review will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would represent significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. A Priority Review means FDA’s goal is to take action on an application within 6 months, compared to 10 months under standard review.

Emcitate® (tiratricol) has previously been granted Rare Pediatric Disease Designation by the FDA and is eligible to receive a Priority Review Voucher (PRV) upon approval, and a PRV was consequently requested in connection with the submission. The transferability of PRVs has created an active secondary market for these vouchers, selling for $150-$205 million per voucher in 2025/26.

https://en.wikipedia.org/wiki/Emetine

Egetis Announces FDA Acceptance and Priority Review of NDA for Emcitate (tiratricol) for MCT8 Deficiency

FDA Approves Nereus (tradipitant) for the Prevention of Motion Sickness

2026-07-17T09:40:56.633+05:30

Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration has approved Nereus (tradipitant), an oral neurokinin-1 (NK-1) receptor antagonist, for the prevention of vomiting induced by motion. This approval marks the first new pharmacologic treatment in motion sickness in over four decades, representing a significant advancement in the understanding and management of this debilitating physiologic response that affects a substantial portion of the population and has long been recognized as a factor affecting military operational readiness.

"This approval underscores the strong scientific evidence in the antiemetic effects of Nereus in motion sickness," said Mihael H. Polymeropoulos, M.D., President, CEO and Chairman of the Board of Vanda Pharmaceuticals. "For the first time in over 40 years, patients have access to a novel therapy grounded in modern neuropharmacology, offering effective prevention without the limitations of existing options. We are proud of this historic milestone and grateful to the Vanda researchers, patients, investigators, and regulators who contributed to this achievement."

The efficacy of Nereus is supported by robust data from three pivotal clinical trials—two Phase 3 real-world provocation studies conducted on boats (Motion Syros and Motion Serifos) and one additional supporting study—with participants who had documented histories of motion sickness. In Motion Syros (n=365), vomiting incidence was 18.3–19.5% with Nereus versus 44.3% with placebo (p<0.0001).1 In Motion Serifos (n=316), vomiting rates were 10.4–18.3% with Nereus versus 37.7% with placebo (p≤0.0014), representing risk reductions of over 50–70%.2 Across the pivotal program, Nereus consistently demonstrated significant reductions in vomiting and a favorable safety profile consistent with acute use.

Motion sickness has been recognized as a critical factor in military operations since World War II, most notably during the D-Day invasion of Normandy in 1944, where severe seasickness impaired the effectiveness of troops, including paratroopers of the 101st Airborne Division deployed in rough Channel crossings and airborne drops. The condition was elevated to a strategic imperative, prompting early research into antiemetic therapies to ensure operational readiness during large-scale troop deployments by sea, air, and land.

Today, motion sickness remains prevalent in civilian life, with approximately 25–30% of adults3 —roughly 65–78 million people in the U.S.—experiencing symptoms during common travel modes such as cars, planes, or boats. Globally, up to one-third of individuals are highly susceptible.4 While most cases are mild, an estimated 5–15% of the population experiences severe, recurrent symptoms that can significantly impact quality of life. This severe segment comprises two key groups: those whose illness is inadequately controlled by existing therapeutic options, leaving them with persistent debilitating symptoms despite treatment, and those whose illness is so severe that it leads to avoidance of engaging in motionprovoking activities altogether, resulting in altered travel plans, missed opportunities, or complete abstention from certain modes of transportation or experiences. Tens of millions seek pharmacologic treatment annually, primarily through over-the-counter options, though many patients opt for prescription therapies when escalating care. Motion sickness arises from a sensory conflict between visual, vestibular, and proprioceptive inputs, triggering the release of substance P and activation of NK-1 receptors in the central nervous system, leading to nausea and vomiting. Nereus's mechanism of action—potent and selective antagonism of NK-1 receptors—directly addresses this pathway.

The approval of Nereus for the prevention of vomiting induced by motion validates its pharmacological profile and paves the way for further exploration of NK-1 antagonism in related vomit-inducing conditions. Vanda is advancing tradipitant in clinical development for gastroparesis, a chronic disorder characterized by delayed gastric emptying and persistent nausea/vomiting, as well as for the prevention of nausea and vomiting induced by GLP-1 receptor agonists—a common side effect impacting adherence in the rapidly growing obesity and diabetes treatment landscape.

Vanda anticipates launching Nereus for the prevention of vomiting induced by motion in the coming months and remains committed to expanding its therapeutic potential across indications driven by substance P-mediated pathways.

https://en.wikipedia.org/wiki/Tradipitant

FDA Approves Nereus (tradipitant) for the Prevention of Motion Sickness

FDA Approves Zycubo (copper histidinate) for Children With Menkes Disease

2026-07-16T09:43:59.506+05:30

Sentynl Therapeutics Inc. (“Sentynl”), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences Limited (“Zydus Group”), announced that the U.S. Food and Drug Administration (“FDA”) has approved Zycubo® (copper histidinate) for the treatment of Menkes disease in pediatric patients. This is the first and only treatment approved for Menkes disease, a rare and fatal genetic condition, in the United States. Zycubo® is not indicated for the treatment of Occipital Horn Syndrome.

“Approval is a pivotal step towards achieving our goal of making a meaningful impact on patients, caregivers, and the rare disease community,” said Dr. Sharvil P. Patel, Managing Director, Zydus Lifesciences Limited. “This milestone marks a transformative moment for the Zydus Group and for families affected by Menkes disease. For the first time, patients have access to an approved therapy, offering hope where no options existed. We are proud to advance patient care and deliver innovative solutions to those who need them most.”

Menkes disease is a rare X-linked recessive pediatric disease caused by mutations of the copper transporter ATP7A encoded by the ATP7A gene. Patients with Menkes disease are born with the inability to absorb dietary copper and subsequently have impaired copper transport across the blood-brain barrier, and, until now, there has been no approved treatment in the United States. Zycubo® is a subcutaneous injectable formulation of copper histidinate that restores copper homeostasis and maintains copper levels in patients with Menkes disease.

“Menkes disease presents significant challenges for patients and their families. With no known cure, most untreated patients do not survive beyond three years of age,” said Matt Heck, CEO, Sentynl. “FDA’s approval serves as compelling affirmation that a safe and effective therapy is now available for patients living with this devastating disease.”

The approval is supported by positive topline clinical efficacy results for Zycubo, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment (“ET”) with Zycubo, compared to an untreated contemporaneous external control cohort, with a nearly 80% reduction in the risk of death. Median overall survival (“OS”) was 177.1 months for Zycubo ET cohort compared to 17.6 months for the untreated contemporaneous external control cohort.

The most common adverse reactions (incidence ≥7%) were pneumonia, viral infection, respiratory failure, seizure, bacterial infection, hemorrhage, hypotension, vomiting, tachycardia, pyrexia, volume depletion, fracture, dyspnea, transaminases elevation, diarrhea, fungal infection, anemia, and local administration reaction.

“This milestone represents the culmination of decades of research into better understanding and ultimately finding an effective treatment for Menkes disease,” said Dr. Stephen Kaler, a clinical genetics and genomics specialist at the Columbia University Medical Center. “Increased awareness of Menkes disease and rapid testing upon suspicion are critical, as beginning copper histidinate therapy in affected neonates has been shown to reduce symptoms and prolong life.”

Acquired from Cyprium Therapeutics in 2023, Sentynl has advanced Zycubo through the final stages of development with the FDA based on positive results from pivotal studies, receiving Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and the FDA Orphan Drug Designations. Copper histidinate has also been granted Orphan Designation by the European Medicines Agency.

ttps://upload.wikimedia.org/wikipedia/commons/thumb/a/ae/Copper_histidinate_v2.svg/250px-Copper_histidinate_v2.svg.png

FDA Approves Zycubo (copper histidinate) for Children With Menkes Disease

2026-07-15T09:30:15.314+05:30

Menkes disease is a rare X-linked recessive pediatric disease caused by mutations of the copper transporter ATP7A encoded by the ATP7A gene. Patients with Menkes disease are born with the inability to absorb dietary copper and subsequently have impaired copper transport across the blood-brain barrier, and, until now, there has been no approved treatment in the United States. Zycubo® is a subcutaneous injectable formulation of copper histidinate that restores copper homeostasis and maintains copper levels in patients with Menkes disease.

FDA Approves Zycubo (copper histidinate) for Children With Menkes Disease

FDA Approves Yuvezzi (carbachol and brimonidine tartrate) Ophthalmic Solution to Treat Presbyopia

2026-07-14T11:56:06.557+05:30

Tenpoint Therapeutics, Ltd., a global, commercial biotechnology company focused on developing groundbreaking treatments to improve vision in the aging eye, today announced that the U.S. Food and Drug Administration (FDA) approved Yuvezzi (carbachol and brimonidine tartrate ophthalmic solution) 2.75%/0.1%, previously known as Brimochol™ PF, the first and only dual-agent eye drop for the treatment of presbyopia in adults. Presbyopia, the gradual loss of near vision that typically begins around age 45, affects about two billion people globally and 128 million people in the U.S.3,4,5 Yuvezzi is expected to be broadly commercially available in the U.S. in Q2 2026.

Carbachol

brimonidine

Yuvezzi (carbachol and brimonidine tartrate ophthalmic solution) 2.75%/0.1% isthe only dual-agent presbyopia-correcting eye drop intentionally designed to deliver durability, tolerability, and safety

Yuvezzi was studied in two pivotal Phase 3 trials enrolling more than 800 patients, including the world’s largest and longest safety study for presbyopia eye drops1,2

Yuvezzi achieves miosis from 30-minutes up to 10 hours with one drop daily1

The FDA approval of Yuvezzi is based on positive data from two Phase 3 studies. The Phase 3 BRIO I study demonstrated a superior benefit of the combination therapy over the individual actives – a requirement for FDA approval of a fixed-dose combination.1 In the second Phase 3 study, BRIO II, which was vehicle-controlled, Yuvezzi achieved all primary near vision improvement endpoints with statistically significant three-lines or greater improvement in binocular uncorrected near visual acuity (BUNVA) over 8 hours, without the loss of one line or more in binocular uncorrected distance visual acuity (BUDVA).2 In addition, Yuvezzi was well-tolerated with no treatment-related serious adverse events observed in the more than 72,000 treatment days monitored in BRIO II, the longest safety study (12 months) conducted in presbyopia to-date.2 The most common side effects of Yuvezzi are headache, impaired vision and temporary eye pain or eye irritation.6

Eye redness was not a commonly reported side effect in clinical trials of Yuvezzi. In BRIO I and BRIO II the reports of adverse events of ocular hyperemia (eye redness) were low.1,2 In BRIO II, the rate of reported adverse events of ocular hyperemia (eye redness) was lower in subjects receiving Yuvezzi (2.8%) than carbochol alone (10.7%).2

“The FDA approval of Yuvezzi represents a significant milestone for the millions of people in the U.S. living with presbyopia and its daily frustrations and challenges,” said Henric Bjarke, Chief Executive Officer of Tenpoint Therapeutics. “As the first FDA-approved dual-agent eye drop for presbyopia, Yuvezzi leverages the mechanisms of carbachol and brimonidine tartrate to deliver sharp near vision with favorable tolerability. People deserve treatments that not only work but also can fit conveniently into their daily lives, and Yuvezzi brings an innovative new option to the presbyopia category. This approval marks the first groundbreaking therapy for Tenpoint Therapeutics aimed at advancing our mission to bring innovation to the aging eye.”

“The impact of presbyopia is often underestimated, and current solutions like glasses, contacts or surgery have fallen short in meeting the real-world needs of people who struggle with close-up tasks,” said John Hovanesian, M.D., FACS, of Harvard Eye Associates in Laguna Hills, California. “Yuvezzi introduces a novel approach by combining carbachol and brimonidine tartrate in a single daily eye drop that sharpens near vision and maintains tolerability throughout the day. Yuvezzi was intentionally designed to deliver both efficacy and tolerability, which represents an important step forward in delivering a complete, noninvasive option for people with presbyopia.”

“Presbyopia remains a universal and progressive condition that requires individualized management strategies,” said Mile Brujic, OD, FAAO, of Premier Vision Group in Bowling Green, Ohio. “I’m excited that I will be able to offer my patients YuvezziTM, the first-of-its-kind eye drop well-suited for long-term use. For people seeking a reliable, lifestyle-friendly solution to manage their near vision challenges in aging eyes, Yuvezzi is a valuable and practical advancement.”

“We’ve been working diligently to bring Yuvezzi to the eye care community and to the millions of adults looking for the optimal solution to address their frustration with presbyopia,” said Carol Kearney, Chief Commercial Officer of Tenpoint Therapeutics. “It was important to us that we provide an option that fits seamlessly into active, social and professional lifestyles, and we intentionally designed Yuvezzi to offer the right balance of efficacy, tolerability and ease of use.”

https://en.wikipedia.org/wiki/Carbachol

https://go.drugbank.com/drugs/DB00484

FDA Approves Yuvezzi (carbachol and brimonidine tartrate) Ophthalmic Solution to Treat Presbyopia

FDA Approves Adquey (difamilast 1%) Ointment for the Treatment of Mild-to-Moderate Atopic Dermatitis

2026-07-13T09:26:24.882+05:30

Acrotech Biopharma Inc., a bio-pharmaceutical company focused on developing and commercializing innovative propriety medications, in collaboration with Otsuka Pharmaceutical Co., Ltd., announced the U.S. Food and Drug Administration (FDA) approval of New Drug Application (NDA) for Adquey (difamilast 1%) ointment, for the topical treatment of mild-to-moderate atopic dermatitis (AD) in adults and pediatric patients aged 2 and older.

Adquey is a novel, non-steroidal, topical phosphodiesterase 4 (PDE4) inhibitor. This approval provides a new non-steroidal, topical treatment option for the millions of Americans living with atopic dermatitis. "The approval of Adquey represents a significant milestone in our commitment to advancing dermatology care," said Ashish Anvekar, President of Acrotech Biopharma Inc. "Patients and clinicians have long sought effective, long-term, non-steroidal treatments that can manage both the inflammation and pruritus associated with eczema. We intend to bring this valuable treatment option to the patients in the U.S. as soon as possible."

Clinical Trial Results

FDA approval was supported by multiple studies including pivotal Phase III controlled trials, which demonstrated that a significantly greater proportion of patients treated with Adquey achieved Investigator’s Global Assessment (IGA) success compared to vehicle (placebo) after four weeks of treatment. The safety profile of Adquey AD was consistent across all three trials. The most common adverse reactions (≥1% and greater than vehicle) was nasopharyngitis. Less common (<1%) adverse reactions in subjects treated with Adquey included application site folliculitis, contact dermatitis, application site rash, and molluscum contagiosum.

https://en.wikipedia.org/wiki/Difamilast

FDA Approves Adquey (difamilast 1%) Ointment for the Treatment of Mild-to-Moderate Atopic Dermatitis

FDA Approves Bysanti (milsaperidone) for the treatment of Bipolar I Disorder and Schizophrenia

2026-07-11T09:59:58.489+05:30

Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has approved Bysanti (milsaperidone) tablets, a first line therapy for the acute treatment of manic or mixed episodes associated with bipolar I disorder and for the treatment of schizophrenia in adults.

Bysanti is a new chemical entity (NCE) that belongs in the class of atypical antipsychotics. In clinical studies Bysanti demonstrated bioequivalence to iloperidone across the therapeutic dosing spectrum enabling it to leverage well-established knowledge of efficacy and safety derived from a rich clinical development program and more than 100,000 patient-years of real-world experience with Fanapt® (iloperidone). As such Bysanti represents a novel therapeutic option with a trusted safety profile in the treatment of these serious psychiatric conditions.

"The Bysanti approval marks a significant step forward, offering patients and providers a reliable new treatment grounded in extensive clinical heritage," said Mihael H. Polymeropoulos, M.D., President, CEO and Chairman of the Board of Vanda Pharmaceuticals. "Bysanti exemplifies a new era of accelerated innovation in drug development that can transform how we address unmet needs in behavioral health."

Bysanti is currently being tested as a once-daily adjunctive treatment in treatment-resistant major depressive disorder in an ongoing clinical study expected to complete by the end of this year.

Bysanti (milsaperidone), a new chemical entity, rapidly interconverts to iloperidone, providing dual active molecules that work in tandem by antagonizing dopamine D2, serotonin 5-HT2A, and alpha1-adrenergic receptors to modulate key pathways in these disorders. Its safety profile aligns closely with that established for iloperidone.

Bysanti's unique in-class receptor binding profile, featuring strong alpha-adrenergic binding in excess of dopamine and serotonin receptor binding, makes it suitable for further investigation in conditions that include symptoms of hostility, agitation, and hyperarousal.

https://en.wikipedia.org/wiki/Milsaperidone

FDA Approves Bysanti (milsaperidone) for the treatment of Bipolar I Disorder and Schizophrenia

Caffeine reversed memory problems caused by sleep deprivation

2026-07-10T09:28:54.717+05:30

In continuation of my update on Caffeine

Researchers at the Yong Loo Lin School of Medicine at the National University of Singapore (NUS Medicine) have found that caffeine can help restore a specific type of memory that is impaired by sleep deprivation. The findings, published in Neuropsychopharmacology, reveal how caffeine acts on a well-defined brain pathway involved in social memory, the ability to recognize and distinguish people we have encountered before.

The research provides new insight into how sleep loss affects the brain and suggests that caffeine's benefits may extend beyond simply increasing alertness.

How Sleep Loss Affects Social Memory

The study was led by Associate Professor Sreedharan Sajikumar and first author Dr. Lik-Wei Wong from the Department of Physiology and the Healthy Longevity Translational Research Program at NUS Medicine.

The team focused on a part of the brain known as the hippocampal CA2 region. The hippocampus is critical for learning and memory, while the CA2 area plays a particularly important role in forming social memories. This brain region also receives signals involved in regulating sleep and wakefulness.

To investigate the effects of sleep deprivation, the researchers subjected laboratory animals to five hours of sleep loss. Afterward, caffeine was provided in drinking water for unrestricted consumption over a seven-day period.

Caffeine Restored Brain Communication

Caffeine is a stimulant that blocks adenosine receptor signaling pathways. Adenosine accumulates during periods of wakefulness and helps reduce brain activity, contributing to feelings of sleepiness.

The researchers then performed electrophysiological recordings on hippocampal tissue samples to assess synaptic plasticity, the brain's ability to strengthen or weaken connections between nerve cells in response to experience and learning.

The results showed that sleep deprivation disrupted the maintenance of synaptic plasticity in the CA2 region. Communication between neurons weakened, reducing the brain's capacity to strengthen important neural connections. These changes were accompanied by noticeable deficits in social recognition memory.

Overall, the findings demonstrated that sleep loss impaired both brain function and behavior through a specific neural circuit.

A Targeted Effect on Memory Circuits

The researchers also found that caffeine administered before sleep deprivation restored synaptic communication in the CA2 region and returned plasticity to normal levels.

As a result, the social memory deficits caused by sleep loss were reversed. Importantly, caffeine's effects were highly selective. Rather than broadly increasing activity throughout the brain, it specifically restored the disrupted pathway linked to social memory.

This targeted action meant that animals in the control group that had not experienced sleep deprivation did not show signs of excessive neural stimulation despite receiving caffeine.

"Sleep deprivation does not just make you tired. It selectively disrupts important memory circuits," noted Dr. Wong. "We found that caffeine can reverse these disruptions at both the molecular and behavioral levels. Its ability to do so suggests that caffeine's benefits may extend beyond simply helping us stay awake."

Assoc Prof Sajikumar added, "Our findings position the CA2 region as a critical hub linking sleep and social memory. This research enhances our understanding towards the biological mechanisms underlying sleep-related cognitive decline. This could inform future approaches to preserving cognitive performance."

Implications for Brain Health and Future Research

The findings highlight the essential role sleep plays in maintaining healthy cognition and memory. By showing that caffeine can restore specific neural pathways affected by sleep deprivation, the study provides new insight into potential targeted approaches for addressing cognitive decline.

The researchers plan to continue investigating how caffeine influences memory consolidation and memory retrieval. Future studies will also use targeted manipulations of brain circuits to better understand the causal relationship between neural pathways and memory function.

https://en.wikipedia.org/wiki/Caffeine

Caffeine reversed memory problems caused by sleep deprivation

FDA Approves Desmoda (desmopressin acetate) Oral Solution for Central Diabetes Insipidus

2026-07-09T14:20:00.381+05:30

In continuation of my update on Desmoda

Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the U.S. Food and Drug Administration (FDA) approval of a New Drug Application (NDA) for Desmoda™ (desmopressin acetate) Oral Solution for the management of central diabetes insipidus, also known as arginine vasopressin deficiency (AVP-D), as antidiuretic replacement therapy for patients of all ages.

Desmoda is the first and only FDA-approved desmopressin oral solution

Commercial launch expected on March 9, 2026

Desmoda eliminates tablet splitting and crushing, enabling precise, individualized dosing for patients of all ages

The product is expected to be a significant long-term growth contributor, with potential peak sales of $30-50 million annually and patent protection extending through 2044

Desmoda is the first and only FDA-approved oral liquid formulation of desmopressin, developed to support precise, individualized dosing in a disease where careful titration is essential to maintaining water balance. Desmoda is supplied as a ready-to-use oral solution (0.05 mg/mL) that does not require tablet splitting, crushing, refrigeration, mixing, or shaking.

Eton estimates that there are more than 13,000 patients with central diabetes insipidus in the U.S., including approximately 3,000-4,000 pediatric patients. The company expects product peak sales of $30-50 million annually.

“Desmoda represents one of the most important product launches in Eton’s history. It builds on our strategy of delivering differentiated therapies to rare endocrine patients. By leveraging our existing pediatric endocrinology platform, we believe Desmoda has the potential to become a foundational therapy in this category,” said Sean Brynjelsen, Chief Executive Officer of Eton Pharmaceuticals. “In central diabetes insipidus, effective long-term management depends on accurately matching desmopressin dosing to each patient’s diurnal pattern of water balance. Historically, clinicians have primarily relied on formulations that were not designed for fine dose adjustments or had other administration issues, and both clinicians and families often had to rely on workarounds. Desmoda introduces a liquid solution designed to deliver individualized dosing precision and consistency for patients across the age spectrum.”

Central diabetes insipidus is a rare, but serious condition caused by inadequate production of the hormone vasopressin from the hypothalamus/posterior pituitary. Treatment with desmopressin is the standard of care, but dosing must be individualized to avoid complications related to over- or under-treatment. Oral liquid dosing allows clinicians to incrementally fine-tune therapy and adjust dosing over time as clinical needs evolve.

“Central diabetes insipidus requires careful, individualized management, where dosing precision and flexibility truly matter. Having a liquid formulation of desmopressin has the potential to meaningfully support how we initiate and manage therapy. It’s encouraging to see innovation that directly addresses real-world challenges faced by clinicians and families and gives greater confidence when tailoring treatment across age groups,” said Dr. Lewis Blevins, Director of the California Center for Pituitary Disorders and Professor of Medicine and Neurological Surgery at the University of California, San Francisco.

“For families living with central diabetes insipidus/AVP-D, treatment demands precision, consistency, and careful management of dosing. A liquid option like Desmoda is a meaningful improvement because it helps make sure that the dose is always right, every time. This is very important in cases where the wrong dose can have a big impact," said Muriël Marks, board member of Worldwide Adrenal and Pituitary Organizations (‘WAPO’).

Desmoda will be promoted by Eton’s existing team of pediatric endocrinology rare disease specialists which currently promotes ALKINDI SPRINKLE® (hydrocortisone), KHINDIVITM (hydrocortisone), and INCRELEX® (mecasermin). Desmoda is expected to be available on March 9th exclusively through Anovo, a specialty pharmacy dedicated to serving patients with rare and chronic conditions. Anovo will administer the Eton Cares Program in partnership with Eton Pharmaceuticals. The program provides prescription fulfillment, insurance benefits investigation, educational support, financial assistance for qualified patients, and other services designed to help patients access treatment. Eton Cares will offer co-pay assistance to allow for $0 co-pay for qualifying patients.

https://en.wikipedia.org/wiki/Desmopressin

FDA Approves Desmoda (desmopressin acetate) Oral Solution for Central Diabetes Insipidus

FDA Grants Accelerated Approval to Yuviwel (navepegritide) for Children with Achondroplasia

2026-07-08T15:11:50.137+05:30

Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has granted approval under the FDA’s Accelerated Approval Program for Yuviwel (navepegritide; developed as TransCon® CNP), the first and only once-weekly treatment indicated to increase linear growth in children 2 years of age and older with achondroplasia with open epiphyses and the only one to provide continuous systemic exposure to CNP over the weekly dosing interval. Continued approval for this indication, which was based on an improvement in annualized growth velocity (AGV), may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

The first and only approved achondroplasia therapy to provide continuous systemic exposure to CNP over the weekly dosing interval
Commercial availability expected during early part of Q2 2026
Rare Pediatric Disease Priority Review Voucher granted in connection with approval

Achondroplasia is a rare genetic condition causing skeletal dysplasia and, for many affected individuals, an increased risk of muscular, neurological, and cardiorespiratory complications. Yuviwel is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia.

“The approval of once-weekly Yuviwel is a major step forward in the treatment of children with achondroplasia, giving physicians for the first time the option of prescribing a once-weekly medicine backed by compelling efficacy and excellent tolerability data from three randomized, double-blind, placebo-controlled clinical trials,” said Carlos A. Bacino, MD, FACMG, Professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital. “My goal is to help children and parents develop care plans tailored to their individual needs and objectives, and I look forward to adding Yuviwel to my discussions with them.”

“Little People of America, the largest national advocacy and support organization for people with dwarfism, is committed to ensuring that the voices of people with dwarfism remain central in conversations about research and medical options such as Yuviwel,” said the Board of Directors of Little People of America. “We champion dwarf and disability pride, advocate for inclusion and respect, and foster open dialogue across diverse perspectives. Our goal is to empower individuals and families to make healthcare decisions that reflect their own values and experiences, while pushing for research efforts and new treatment options such as this that could have the potential to support outcomes that truly matter to our community.”

The FDA based its approval of Yuviwel on their review of the clinical package for TransCon CNP submitted with the Company’s New Drug Application, which included safety and efficacy data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data. The pivotal ApproaCH Trial data is available in JAMA Pediatrics.i

“We are confident in Yuviwel’s potential to transform the treatment of achondroplasia and are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. “We have listened to advocacy groups for people with dwarfism to ensure we address what the community actually cares about. This reflects our ongoing commitment to pursue outcomes that patient communities have told us are important to them, and gives the achondroplasia community a new way to look at the promise of pharmacological treatment options.”

Ascendis expects to make Yuviwel available through prescribing physicians in the United States during the early part of the second quarter of 2026. Ascendis plans to offer a suite of patient services for Yuviwel through its U.S. Ascendis Signature Access Program (A.S.A.P.), including support navigating the treatment journey and financial assistance programs for eligible patients.

With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. This program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.

FDA Grants Accelerated Approval to Yuviwel (navepegritide) for Children with Achondroplasia

FDA Approves Icotyde (icotrokinra) for the Treatment of Plaque Psoriasis

2026-07-07T09:33:57.856+05:30

Johnson & Johnson (NYSE: JNJ) announced today that the U.S. Food and Drug Administration (FDA) has approved Icotyde (icotrokinra), an interleukin-23 (IL-23) receptor antagonist for the treatment of moderate-to-severe plaque psoriasis in adults and pediatric patients 12 years of age and older who weigh at least 40 kg who are candidates for systemic therapy or phototherapy. Icotyde is the first and only targeted oral peptide that precisely blocks the IL-23 receptor.

Johnson & Johnson introduces the first and only IL-23R targeted oral peptide that delivers complete skin clearance and favorable safety profile in a once-daily pill
Icotyde offers an innovative new option for patients with moderate-to-severe plaque psoriasis to address patients cycling on topical therapies in need of systemic treatment

“Icotyde delivers something unique in psoriasis treatment – combining skin clearance with a favorable safety profile in a once‑daily pill, making it an easy addition to a patient’s routine,” said Linda Stein Gold, M.D., Director of Dermatology Clinical Research at Henry Ford Health.a “With new guidance from the International Psoriasis Council that clarifies when to move beyond cycling on topical treatments to systemic therapy, an innovative option like Icotyde is a potential game‑changer for many adult and adolescent patients.”

Clinical evidence summary

Icotyde met all primary efficacy endpoints and demonstrated a favorable safety profile across four Phase 3 studies including 2,500 patients. The approval is based on an unprecedented body of evidence from the ICONIC clinical development program, which simultaneously evaluated Icotyde in adults and adolescents, high impact sites such as scalp and genital PsO, and in duplicate head-to-head trials versus an active comparator. In the head-to-head superiority studies, approximately 70% of patients achieved clear or almost clear skin (IGA 0/1) and 55% of patients achieved a Psoriasis Area and Severity Index (PASI) 90 response at Week 16.3,b,c Rates of adverse reactions for Icotyde treated patients were within 1.1% of placebo through Week 16 and no new safety signals were identified through Week 52.

“With the FDA approval of Icotyde, Johnson & Johnson is setting a new standard for the treatment of moderate-to-severe plaque psoriasis,” said Jennifer Taubert, Executive Vice President, Worldwide Chairman, Innovative Medicine, Johnson & Johnson. “We’re proud to bring this game-changing innovation to the market, marking a transformative shift in plaque psoriasis management that empowers patients and clinicians to reach their treatment goals.

Unmet need in moderate-to-severe plaque psoriasis

Psoriasis affects more than 8 million Americans, impacting physical comfort and quality of life, especially when lesions are on visible or sensitive areas.5 For many with moderate-to-severe disease, targeted systemic treatments are key. This aligns with International Psoriasis Council guidance to transition to systemic therapy if two cycles of topical medications applied for four weeks fail to bring meaningful improvement.

“Finding the right treatment can take time, during which people with psoriatic disease should be considering multiple factors from efficacy to safety to how the treatment fits into their everyday life,” said Leah M. Howard, J.D., President and CEO of the National Psoriasis Foundation.d “The approval of a novel systemic therapy changes the conversation about treatment options for our community.”

“The approval of Icotyde represents a pivotal moment for people with plaque psoriasis,” said John Reed, M.D., Ph.D., Executive Vice President, R&D, Innovative Medicine, Johnson & Johnson. “At Johnson & Johnson, we are harnessing our scientific expertise to transform cutting-edge science into meaningful solutions for patients. Icotyde is a fundamentally different treatment with the potential to redefine what physicians and patients can expect from psoriasis treatment.

https://en.wikipedia.org/wiki/Icotrokinra

FDA Approves Icotyde (icotrokinra) for the Treatment of Plaque Psoriasis

FDA Approves Lynavoy (linerixibat) for Cholestatic Pruritus in Patients with Primary Biliary Cholangitis

2026-07-06T10:20:44.161+05:30

GSK announced the US Food and Drug Administration (FDA) approval of Lynavoy (linerixibat) for the treatment of cholestatic pruritus in adult patients with PBC. Lynavoy, an ileal bile acid transporter (IBAT) inhibitor that reduces multiple drivers of chronic itch, is the first medicine approved in the US for this indication.5

Lynavoy, an ileal bile acid transporter (IBAT) inhibitor, is the first medicine approved in the US for the treatment of cholestatic pruritus in patients with PBC

Up to 89% of people living with PBC experience cholestatic pruritus, an internal itch with a debilitating impact on quality of life1-4

Approval based on the positive GLISTEN phase III trial with regulatory reviews underway in the EU, UK, Canada and China

GSK previously announced on 9 March a licence agreement under which Alfasigma S.p.A. will acquire worldwide exclusive rights to develop, manufacture and commercialise linerixibat. This transaction is ongoing and is subject to customary conditions, including applicable regulatory agency clearances such as under the Hart-Scott-Rodino Act in the US.

Cholestatic pruritus is an internal itch experienced by up to 89% of people living with PBC, a rare autoimmune disease that can lead to liver failure.1-4 It is a serious condition that can be debilitating, with patients experiencing sleep disturbance, fatigue, impaired quality of life and even sometimes requiring liver transplantation in the absence of liver failure

https://en.wikipedia.org/wiki/Linerixibat

FDA Approves Lynavoy (linerixibat) for Cholestatic Pruritus in Patients with Primary Biliary Cholangitis

FDA Approves Lifyorli (relacorilant) Plus Nab-Paclitaxel for the Treatment of Patients with Platinum-Resistant Ovarian Cancer

2026-07-03T15:01:19.436+05:30

In continuation of my update on relacorilant

Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, announced the U.S. Food and Drug Administration approval of Lifyorli (relacorilant) in combination with nab-paclitaxel for the treatment of adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens, at least one of which included bevacizumab. Lifyorli is the first FDA-approved selective glucocorticoid receptor antagonist (SGRA).

Approval was based on the positive outcomes of Lifyorli's pivotal ROSELLA trial, which enrolled 381 patients with platinum-resistant ovarian cancer who had received one to three prior lines of therapy, at least one of which included bevacizumab. Patients were randomized 1:1 to receive either Lifyorli plus nab-paclitaxel or nab-paclitaxel monotherapy. No biomarker selection was required.

ROSELLA met its dual primary endpoints of progression-free and overall survival. Patients treated with Lifyorli in addition to nab-paclitaxel experienced a 35 percent reduction in the risk of death compared to patients treated with nab-paclitaxel alone (hazard ratio: 0.65; p-value: 0.0004), with a median overall survival (OS) of 16.0 months, compared to 11.9 months for patients receiving nab-paclitaxel alone, a difference of 4.1 months. Patients who received Lifyorli in addition to nab-paclitaxel also experienced a 30 percent reduction in the risk of disease progression (hazard ratio: 0.70; p-value: 0.008), as assessed by blinded independent central review (PFS-BICR), compared to patients treated with nab-paclitaxel alone.

The combination of Lifyorli with nab-paclitaxel was well-tolerated and manageable. The safety of Lifyorli was assessed in a pooled analysis of patients from ROSELLA and Lifyorli's Phase 2 trial. The prescribing information for Lifyorli includes warnings and precautions for neutropenia and severe infections, adrenal insufficiency, exacerbation of conditions treated with glucocorticoids and embryo-fetal toxicity. The most common adverse reactions experienced by more than 20 percent of patients (including laboratory abnormalities) were decreased hemoglobin, decreased neutrophils, fatigue, nausea, diarrhea, decreased platelets, rash and decreased appetite.

Data from ROSELLA were first presented at ASCO 2025 (American Society of Clinical Oncology) with simultaneous publication in The Lancet. Complete results from ROSELLA will be presented at the Society of Gynecologic Oncology (SGO) meeting in April.

"Data demonstrate that Lifyorli plus nab-paclitaxel provides a clinically meaningful benefit in overall survival for patients with platinum-resistant ovarian cancer and is well tolerated. Lifyorli is positioned to become a new standard-of-care treatment," said Rob Coleman, M.D., Texas Oncology and special advisor to the president of the GOG Foundation. "Having a new treatment for this advanced, recurrent disease will provide clinicians with a compelling option to help patients with this extremely difficult-to-treat cancer."

"The FDA's approval of Lifyorli in combination with nab-paclitaxel is welcome news to all of us in the ovarian cancer community," said Sarah DeFeo, Chief Program Officer at Ovarian Cancer Research Alliance (OCRA). "We are grateful to everyone who participated in the clinical trials, their families and the physicians who helped advance this urgently needed treatment option for patients with platinum-resistant ovarian cancer."

https://en.wikipedia.org/wiki/Relacorilant

FDA Approves Lifyorli (relacorilant) Plus Nab-Paclitaxel for the Treatment of Patients with Platinum-Resistant Ovarian Cancer

How a cheap, century-old drug can improve life with type 1 diabetes

2026-07-02T11:10:19.989+05:30

In continuation of my update on Metformin

A Garvan-led clinical trial has found that using a common and inexpensive type 2 diabetes drug reduces insulin needs in type 1 diabetes, opening doors for improved management of the condition.

For years, doctors have prescribed metformin, an old but common type 2 diabetes medication, to treat insulin resistance in type 1 diabetes. This has been largely based on anecdotal evidence. Now, a clinical trial led by the Garvan Institute of Medical Research has found that metformin does not counteract insulin resistance in type 1 diabetes, but instead reduces the amount of insulin needed to maintain blood sugar levels in the ideal range.

Published in Nature Communications, these surprising findings could improve how doctors manage type 1 diabetes and ease the significant burden that people with the condition face when using insulin alone.

Can we overcome insulin resistance in type 1 diabetes?

Type 1 diabetes is an autoimmune condition affecting over 130,000 Australians in which the immune system incorrectly attacks the insulin-producing cells of the pancreas. As a result, people with type 1 diabetes need to administer insulin for the rest of their lives to regulate their blood sugar levels. Managing blood sugar levels with insulin is not easy. In fact, it is estimated that people living with type 1 diabetes have to make 180 extra decisions per day related to their diabetes management.

In some people with type 1 diabetes, long-term insulin use can lead to insulin resistance, where the body's cells no longer respond effectively to the drug. This means that people need ever-increasing amounts of insulin to keep blood sugar levels under control.

"Insulin resistance is a growing problem in type 1 diabetes. Not only does it make regulating blood sugar levels difficult, but it is an underappreciated risk factor for heart disease, which is one of the biggest causes of health complications and deaths in those with type 1 diabetes," says Dr. Jennifer Snaith, endocrinologist and co-lead of the study.

To address this, a Garvan team led by Dr. Snaith and Professor Jerry Greenfield undertook the world's first randomized controlled trial in adults testing whether metformin, a cheap, oral drug normally used to counteract insulin resistance in type 2 diabetes, could do the same in type 1 diabetes. It is estimated that the drug is currently used off-label by up to 13,000 Australians with type 1 diabetes, but it remains unclear how exactly it works. This trial was called the Insulin Resistance in type 1 Diabetes Managed with Metformin (INTIMET) study.

"We randomized 40 adults with long-term type 1 diabetes to take either metformin or a placebo for six months. We examined whether their insulin resistance changed over that time through a sophisticated and comprehensive research technique, called a clamp study, that allowed us to map insulin resistance in different parts of the body," explains Professor Greenfield.

A surprise finding challenges assumption of metformin action

Unexpectedly, the team found that the use of metformin did not lead to improvements in insulin resistance or changes to blood sugar levels. This suggests that, unlike with type 2 diabetes, metformin does not work to counter insulin resistance in type 1 diabetes. However, metformin did decrease the amount of insulin people needed to keep their blood sugars stable.

"Although we didn't find changes to insulin resistance from the use of metformin, we did show that people taking it used around 12% less insulin than those on placebo. This is an important result. Insulin is a relatively old treatment which, while lifesaving, comes with significant mental and physical burden.

"This means that lowering the amount of insulin used is a priority for many people living with type 1 diabetes. We have shown that a very cheap, accessible medication may serve this purpose and this is very exciting," Dr. Snaith says.

A role for gut microbes?

The team is now investigating how metformin may work to lower the amount of insulin needed by those with type 1 diabetes.

Professor Greenfield explains, "Metformin has been available in various forms for around 100 years, but its mechanism of action remains unknown. We would have expected that the observed reductions in insulin dose induced by metformin in our study would be due to the body becoming more sensitive to insulin, that is, becoming less insulin resistant. But we have shown that is not the case. Our priority is now working out how metformin is achieving this effect."

"There is increasing evidence suggesting that metformin may act on the gut. This is why we are now investigating how metformin changes gut flora, also known as the microbiome, in people with type 1 diabetes. This has not been studied before in type 1 diabetes. We're hoping this will provide clues on metformin's mechanism of action, so that it can be more widely used in the management of type 1 diabetes," adds Dr. Snaith.

How a cheap, century-old drug can improve life with type 1 diabetes