Med-Chemist

Eylea HD (aflibercept) Approved by FDA for the Treatment of Macular Edema Following Retinal Vein Occlusion (RVO) and for Monthly Dosing Across Approved Indications

2026-06-13T10:54:10.723+05:30

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced the U.S. Food and Drug Administration (FDA) approval of Eylea HD (aflibercept) Injection 8 mg for the treatment of patients with macular edema following retinal vein occlusion (RVO) with up to every 8-week dosing after an initial monthly dosing period. The FDA also approved an every 4-week (monthly) dosing option for some patients who may benefit from resuming this dosing schedule across approved indications: wet age-related macular degeneration (wAMD), diabetic macular edema (DME), diabetic retinopathy (DR) and RVO.

Aflibercept is a combination of fluorouracil, leucovorin, and irinotecan,

flurouracil

Folinic acid,

Irinotecan

"We believe these approvals further position Eylea HD as a treatment of choice for certain retinal diseases and underscore our relentless commitment to meeting the needs of patients and the retina specialists who treat them,” said George D. Yancopoulos, M.D., Ph.D., co-Founder, Board Co-Chair, President and Chief Scientific Officer, at Regeneron. “Eylea HD is the first treatment for retinal vein occlusion that can potentially cut the number of injections that patients receive in half compared to existing therapies. And with the addition of a monthly dosing option for all four approved Eylea HD indications, physicians now have greater flexibility and optionality to tailor treatment to meet individual patient needs.”

The FDA approval for the treatment of RVO is based on data from the Phase 3 QUASAR trial that evaluated the efficacy and safety of Eylea HD compared to Eylea® (aflibercept) Injection 2 mg in patients with RVO. QUASAR met its primary endpoint at 36 weeks, with Eylea HD patients dosed every 8 weeks (after either 3 or 5 monthly doses) achieving non-inferior visual acuity gains compared to those receiving Eylea dosed every 4 weeks. The Eylea HD results were consistent across patients with branch retinal vein occlusions, and those with central retinal or hemiretinal vein occlusions. In RVO, the most common adverse reactions reported in ≥3% of patients treated with Eylea HD were intraocular pressure increased, vision blurred, cataract, conjunctival hemorrhage, ocular discomfort/eye pain/eye irritation and vitreous detachment.

In regard to the Eylea HD pre-filled syringe (PFS), Regeneron continues to coordinate with Catalent Indiana, LLC (part of Novo Nordisk A/S) as it works to resolve the outstanding issues identified from a July 2025 FDA general site inspection (not specific to Eylea HD). As previously disclosed, Regeneron also plans to submit to the FDA an application to include an alternate PFS manufacturing filler for the Eylea HD BLA by January 2026.

Eylea HD is approved with dosing intervals from every 8 to 16 weeks for patients with wAMD and DME (following 3 initial monthly doses), every 8 to 12 weeks for patients with DR (following 3 initial monthly doses), and every 8 weeks for patients with RVO (following 3 to 5 initial monthly doses). In clinical trials, some Eylea HD patients did not maintain a response with extended dosing intervals after successful response to initial monthly doses; these patients may benefit from resuming every 4-week dosing.

https://en.wikipedia.org/wiki/Aflibercept#:~:text=Aflibercept%20is%20a%20recombinant%20fusion%20protein%20consisting%20of%20vascular%20endothelial,of%20the%20human%20IgG1%20immunoglobulin.

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

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

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

Eylea HD (aflibercept) Approved by FDA for the Treatment of Macular Edema Following Retinal Vein Occlusion (RVO) and for Monthly Dosing Across Approved Indications

Ofirnoflast (HT-6184) Receives Orphan Drug Designation from U.S. FDA for Myelodysplastic Syndromes

2026-06-11T08:31:39.580+05:30

Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company pioneering therapies that target the root causes of inflammation-driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational medicine ofirnoflast (HT-6184) for the treatment of Myelodysplastic Syndromes (MDS) — a group of bone marrow disorders characterized by ineffective blood cell production and a risk of progression to acute myeloid leukemia (AML).

The FDA grants Orphan Drug Designation to therapies intended for the treatment, prevention, or diagnosis of rare diseases or conditions that affect fewer than 200,000 people in the United States at the time of designation.

"This designation underscores the potential of our approach in Myelodysplastic Syndromes and supports our commitment to developing new treatment options for patients living with MDS," said David Bearss, PhD, Chief Executive Officer of Halia Therapeutics. "Ofirnoflast represents a first-in-class approach to modulating inflammasome biology, an upstream driver of inflammation, with the goal of restoring healthy bone marrow function."

Ofirnoflast is a selective NEK7 allosteric modulator designed to prevent the formation and promote the disassembly of the NLRP3 inflammasome, a central driver of chronic inflammation in multiple diseases. In MDS, inflammasome activation is increasingly recognized as a key contributor to ineffective hematopoiesis and bone marrow failure. By modulating NEK7, ofirnoflast aims to restore immune balance and improve blood-cell production without broad immunosuppression.

"Inflammasome biology represents a promising frontier for hematologic innovation," said Alan F. List, MD, member of Halia Therapeutics' Scientific Advisory Board and former President and CEO of Moffitt Cancer Center. "Ofirnoflast's approach is distinctive in that it seeks to modulate the underlying inflammatory drivers of MDS rather than just its downstream effects. This strategy has the potential to redefine how inflammation-linked bone marrow failure is treated."

Under the FDA's Orphan Drug Act, orphan-drug status provides several incentives, including tax credits for qualified clinical testing, exemption from FDA user fees, and potential for seven years of U.S. market exclusivity upon approval. The FDA also administers grant programs to support clinical research and advance the development of therapies for rare diseases.

About Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes are a group of bone marrow disorders characterized by defective blood-cell formation, leading to anemia, infection risk, and bleeding complications. MDS primarily affects older adults and can progress to acute myeloid leukemia (AML). Current therapies, including hypomethylating agents and growth factors, often provide limited benefit and do not address the underlying inflammatory biology of the disease.

About Ofirnoflast (HT-6184)

Ofirnoflast (HT-6184) is Halia Therapeutics' lead investigational compound and a first-in-class NEK7 modulator that regulates activation of the NLRP3 inflammasome — an upstream molecular complex involved in chronic inflammation. The drug is currently being evaluated across multiple disease areas, including:

Myelodysplastic Syndromes (MDS) – completed Phase 2 study evaluating safety and hematologic outcomes
Obesity (in combination with semaglutide) – ongoing Phase 2 study targeting adipose inflammation and metabolic dysregulation
Alzheimer's Disease – early-stage program focused on genetically at-risk populations

About Halia Therapeutics, Inc.

Halia Therapeutics is a clinical-stage biopharmaceutical company developing therapies that leverage genetic resilience to restore the body's natural ability to resolve inflammation. By targeting the NEK7–NLRP3 inflammasome axis, Halia's pipeline addresses a spectrum of chronic inflammatory and degenerative diseases, including hematologic disorders, metabolic disease, and neurodegeneration.

https://www.guidetomalariapharmacology.org/GRAC/LigandDisplayForward?ligandId=13227

Ofirnoflast (HT-6184) Receives Orphan Drug Designation from U.S. FDA for Myelodysplastic Syndromes - Drugs.com MedNews

Genentech’s Fenebrutinib Shows Unprecedented Positive Phase III Results as the Potential First and Only BTK Inhibitor in Both Relapsing and Primary Progressive Multiple Sclerosis

2026-06-08T09:19:25.735+05:30

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the first Phase III (FENhance 2) of two pivotal, similarly-designed Phase III studies (FENhance 1 and 2) in patients with relapsing multiple sclerosis (RMS) met its primary endpoint. Fenebrutinib, an investigational Bruton’s tyrosine kinase (BTK) inhibitor, significantly reduced the annualized relapse rate (ARR) compared to teriflunomide over a period of at least 96 weeks of treatment.

Additionally, the Phase III FENtrepid pivotal study evaluating fenebrutinib, compared with Ocrevus® (ocrelizumab) in patients with primary progressive multiple sclerosis (PPMS), met its primary endpoint. The results showed that fenebrutinib was non-inferior compared to ocrelizumab, the only approved therapy in PPMS, as measured by a delay in the onset of composite confirmed disability progression over a period of at least 120 weeks of treatment. A numerical benefit for fenebrutinib compared to ocrelizumab was seen as early as week 24, and lasted throughout the observation period.

“Fenebrutinib substantially reduced the number of relapses in RMS and slowed disability progression in PPMS. These unprecedented results suggest that fenebrutinib could potentially become a best-in-disease medicine as the first high-efficacy, oral treatment for people with RMS or PPMS,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. "Therefore, these pivotal results for fenebrutinib may offer new hope for people living with MS, and they reaffirm our enduring commitment to the MS community.”

Liver safety was consistent with previous fenebrutinib studies. Additional safety data is being further evaluated. The results of the second RMS Phase III trial (FENhance 1) are expected by the first half of 2026.

Fenebrutinib targets cells in the immune system known as B cells and microglia. Targeting B cells helps control the acute inflammation that causes relapses, while targeting microglia inside the brain addresses the chronic damage that is thought to drive long-term disability progression. Fenebrutinib, a non-covalent BTKi, is designed to have high potency, selectivity and reversibility. This design allows it to act throughout the body, and also to cross the blood-brain barrier into the central nervous system (CNS) targeting chronic inflammation.

About the FENhance 1 and 2 studies

FENhance 1 and 2 are similarly designed Phase III multicenter, randomized, double-blind, double-dummy, parallel-group studies to evaluate the efficacy and safety of investigational fenebrutinib compared with teriflunomide in a total of 1,497 adult patients with RMS. Eligible participants were randomized 1:1 to receive treatment with either oral fenebrutinib twice a day (and placebo matched to oral teriflunomide once a day) or oral teriflunomide once a day (and placebo matched to oral fenebrutinib twice a day) for at least 96 weeks.

The primary endpoint is annualized relapse rate (ARR). Key secondary endpoints include the time to onset of composite 24-week confirmed disability progression (cCDP24), 12-week confirmed disability progression (CDP12) and 24-week confirmed disability progression (CDP24).

Following the double-blind treatment period, patients have the option to enter an open-label extension (OLE) phase, in which all patients receive treatment with fenebrutinib.

About the FENtrepid study

FENtrepid is a Phase III multicenter, randomized, double-blind, double-dummy, parallel-group study to evaluate the efficacy and safety of fenebrutinib compared with Ocrevus in 985 adult patients with PPMS. Eligible participants were randomized 1:1 to receive treatment with either daily oral fenebrutinib (and placebo matched to intravenous [IV] Ocrevus) or IV Ocrevus (and placebo matched to oral fenebrutinib) for at least 120 weeks.

The primary endpoint is the time to onset of 12-week composite confirmed disability progression (cCDP12). The cCDP incorporates three measures of disability – total functional disability measured by the Expanded Disability Status Scale (EDSS), walking speed measured by the timed 25-foot walk (T25FW), and upper limb function measured by the nine-hole peg test (9HPT). This comprehensive composite endpoint offers greater sensitivity than the EDSS alone, capturing additional aspects of disability and often earlier. Key secondary endpoints include the time to onset of 24-week composite confirmed disability progression (cCDP24), 12-week confirmed disability progression (CDP12) and 24-week confirmed disability progression (CDP24).

Following the double-blind treatment period, patients have the option to enter an open-label extension (OLE) phase, in which all patients receive treatment with fenebrutinib.

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

Genentech’s Fenebrutinib Shows Unprecedented Positive Phase III Results as the Potential First and Only BTK Inhibitor in Both Relapsing and Primary Progressive Multiple Sclerosis - Drugs.com MedNews

U.S. Food and Drug Administration Accepts Bristol Myers Squibb's New Drug Application for Iberdomide in Patients with Relapsed or Refractory Multiple Myeloma

2026-06-06T10:15:46.725+05:30

Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for iberdomide combined with standard treatment (daratumumab + dexamethasone - IberDd) in patients with relapsed or refractory multiple myeloma (RRMM). Iberdomide is part of an investigational, new class of medicines called cereblon E3 ligase modulator (CELMoD) agents. The FDA has granted a Prescription Drug User Fee Act (PDUFA) date of August 17, 2026 for this indication.

Iberdomide has the potential to be the first approved CELMoD agent
The U.S. FDA has granted Breakthrough Therapy Designation and Priority Review for this indication and assigned a target action date of August 17, 2026

“The FDA’s acceptance of this application is a testament to the potential of iberdomide, in combination with anti-CD38 monoclonal antibodies, as a novel, potent, oral treatment option, with a manageable safety profile, for patients with multiple myeloma,” said Cristian Massacesi, executive vice president and chief medical officer, Bristol Myers Squibb. “Furthermore, our filing for iberdomide based on the MRD endpoint, underscores our commitment to pioneering new ways of advancing life-saving therapies for patients living with cancer.”

The filing was based on results from a planned analysis of MRD negativity rates in the Phase 3 EXCALIBER-RRMM study evaluating iberdomide as a treatment for RRMM patients. The EXCALIBER-RRMM trial is ongoing and patients continue to be evaluated for progression-free survival (PFS).

The FDA also granted Breakthrough Therapy designation for iberdomide based on these data.

This review is being conducted under the FDA’s Project Orbis initiative, which enables concurrent review by the health authorities in several other countries.

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

U.S. Food and Drug Administration Accepts Bristol Myers Squibb's New Drug Application for Iberdomide in Patients with Relapsed or Refractory Multiple Myeloma

Deciphera Pharmaceuticals Announces U.S. Food and Drug Administration Acceptance for Filing of New Drug Application for Tirabrutinib in Patients with Relapsed or Refractory PCNSL

2026-06-05T10:25:10.549+05:30

Deciphera Pharmaceuticals, a member of Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”), today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) under the accelerated approval pathway for tirabrutinib, a highly selective irreversible, second generation Bruton tyrosine kinase inhibitor, for the treatment of relapsed or refractory primary central nervous system lymphoma (R/R PCNSL). The FDA has set an action date of December 18, 2026, under the Prescription Drug User Fee Act (PDUFA).

Regulatory submission is based on positive results from the Phase 2 PROSPECT Study

FDA sets PDUFA Date of December 18, 2026

“R/R PCNSL is a rare and aggressive form of non-Hodgkin lymphoma with particularly poor clinical outcomes. Patients often experience difficulty and delay in diagnosis, and once they are diagnosed, there is a high unmet need for a treatment with a favorable safety profile,” said Matthew L. Sherman, M.D., Chief Medical Officer of Deciphera. “The FDA’s acceptance of tirabrutinib’s NDA for filing is an exciting milestone as it brings us one step closer to our goal of providing patients with R/R PCNSL an important new treatment option.”

“We are very pleased that the NDA for tirabrutinib has been accepted for filing,” said Toichi Takino, President and COO of Ono Pharmaceutical Co., Ltd. “This is an important milestone on the way to expanding our commercial pipeline and achieving our goal of becoming a global specialty pharma. Tirabrutinib’s potential to address unmet patient needs embodies our corporate philosophy and we will continue to focus on developing and delivering innovative medicines to benefit patients worldwide.”

The NDA is supported by the positive results from the Phase 2 PROSPECT study, presented at the 2025 American Society for Clinical Oncology (ASCO) Annual Meeting, in which tirabrutinib demonstrated an overall response rate of 67%, a complete response rate of 44%, and a manageable safety profile. If approved, tirabrutinib will be the first BTK inhibitor therapy commercially available in the U.S. for the treatment of patients with R/R PCNSL, and the third commercial therapy for Ono group available in the U.S. The study is currently recruiting patients with R/R PCNSL in a global Phase 3 randomized trial, which will serve as a confirmatory study for this indication (ClinicalTrials.gov NCT07104032.)

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

Deciphera Pharmaceuticals Announces U.S. Food and Drug Administration Acceptance for Filing of New Drug Application for Tirabrutinib in Patients with Relapsed or Refractory PCNSL

FDA Grants Orphan Drug Designation for Tinostamustine in Malignant Glioma

2026-06-04T09:56:28.317+05:30

Purdue Pharma L.P. (“Purdue”) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its pipeline drug tinostamustine for the treatment of malignant gliomas, a broad category of brain and spinal cord cancers that affect both adults and children and includes rapidly growing, invasive tumors like glioblastoma. As many as 22,000 people are diagnosed with malignant gliomas annually in the United States1. FDA grants ODD status to encourage development of promising medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

Purdue is currently investigating tinostamustine in patients with glioblastoma, the most common malignant glioma, that is aggressive, very challenging to treat and for which there is no cure.2 Most patients do not survive more than 15 months with current treatment approaches.3 The ODD request for tinostamustine was supported by robust epidemiologic, clinical and pre-clinical evidence, as well as mechanistic rationale supporting its potential to address a critical unmet medical need for glioblastoma.

“As many as 15,000 people in the U.S. are diagnosed with glioblastoma each year.3 Unfortunately, there is limited survival benefit with existing treatment options,” said Julie Ducharme, Vice President and Chief Scientific Officer, Purdue. “This recognition from FDA is an important milestone in our mission of advancing innovative science in areas of serious, unmet medical need. We look forward to further investigating tinostamustine, which has shown promise in early trials.”

Orphan drug designation is intended to facilitate drug development for rare diseases and may provide certain incentives to drug developers.4,5 These benefits include tax credits for qualified clinical trials, exemption from user fees including New Drug Application (NDA), and a potential for seven years of market exclusivity following approval.

Tinostamustine is a potential first-in-class, new chemical entity that combines two potentially synergistic mechanisms of action, bifunctional alkylating activity and pan histone deacetylase inhibition (or HDAC inhibition). Tinostamustine has the potential to be a first-line treatment for glioblastoma.

“Behind every designation like this are real people, patients and families, facing the devastating reality of malignant gliomas, especially glioblastoma,” said Craig Landau, MD, President and CEO, Purdue Pharma. “We are deeply committed to pursuing this medicine that has the potential to bring hope where few options exist today. Tinostamustine represents a step forward in our efforts to help address the urgent and unmet needs of those affected by these aggressive cancers.”

The Company also recently entered into an agreement with the Global Coalition for Adaptive Research (GCAR) to pursue the Phase 2/3 clinical development of tinostamustine in GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment – NCT03970447), a global adaptive platform trial for glioblastoma.

This press release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that tinostamustine will successfully complete development or gain FDA approval.

https://pubchem.ncbi.nlm.nih.gov/compound/Tinostamustine

FDA Grants Orphan Drug Designation for Tinostamustine in Malignant Glioma - Drugs.com MedNews

Icotrokinra Beneficial for Adults, Teens With Plaque Psoriasis

2026-06-03T09:45:06.871+05:30

For adults and adolescents with moderate-to-severe plaque psoriasis, selective blockade of the interleukin-23 receptor with the targeted oral peptide icotrokinra yields a significantly higher incidence of skin clearance at week 16 than placebo, according to a study published in the Nov. 6 issue of the New England Journal of Medicine.

Robert Bissonnette, M.D., from Innovaderm Research in Montreal, and colleagues conducted a phase 3, randomized trial involving adults and adolescents (aged 12 years and older) with moderate-to-severe plaque psoriasis. Participants were randomly assigned to receive either icotrokinra (200 mg once daily through week 24) or placebo through week 16 followed by transition to icotrokinra (456 and 228 patients, respectively).

The researchers found that 65 percent of the participants receiving icotrokinra and 8 percent of those receiving placebo had an Investigator's Global Assessment (IGA) score of 0 or 1 with ≥2-point reduction from baseline at week 16, and 50 and 4 percent, respectively, had a ≥90 percent reduction from baseline in the Psoriasis Area and Severity Index (PASI) score. At week 16, complete clearance of skin was significantly more likely with icotrokinra than placebo (IGA score 0: 33 versus 1 percent; 100 percent reduction from baseline in the PASI score: 27 versus 1 percent). In each group, 49 percent of patients had at least one adverse event through week 16.

"Once-daily icotrokinra, a systemic targeted oral peptide binding to the interleukin-23 receptor, was effective for treating plaque psoriasis in adults and in adolescents, an age group with limited systemic treatment options," the authors write.

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

Icotrokinra Beneficial for Adults, Teens With Plaque Psoriasis - Drugs.com MedNews

FDA Approves Clotic (clotrimazole otic solution), First Treatment with Clinical Evidence for Fungal Ear Infections

2026-06-02T11:20:54.591+05:30

In continuation of my update on clotrimazole

Laboratorios Salvat, S.A. ("Salvat") announced today that the U.S. Food and Drug Administration (FDA) has approved Clotic (clotrimazole otic solution), the first and only treatment with clinical evidence specifically indicated for fungal otitis externa (otomycosis).

About Clotic

Clotic is a broad-spectrum otic antimycotic agent containing clotrimazole, an azole antifungal that inhibits fungal enzyme production essential for survival. The medication works by blocking cytochrome P450-dependent enzymes, damaging fungal cell walls and disrupting growth, ultimately causing cell death. This preservative-free formulation is administered as ear drops using innovative Blow-Fill-Seal technology in single-dose vials.

Clotic is indicated for the treatment of fungal otitis externa (otomycosis) in adults 18 years of age and older, specifically targeting infections caused by Aspergillus species and Candida species. The treatment is approved only for patients with intact tympanic membranes.

The FDA approval of Clotic was based on comprehensive clinical data demonstrating well-recognized safety and efficacy in treating pathogenic dermatophytes, yeasts, and several species of fungus affecting the outer ear canal. The approval package included analytical and clinical evidence supporting Clotic's effectiveness as the first clinically proven treatment option for otomycosis.

Clotic is administered twice daily for 14 days, with each single-dose vial (1%/0.17mL) delivering the complete treatment dose. The innovative packaging protects the preservative-free formulation from humidity and contamination while ensuring precise dosing.

The approval of Clotic marks an important milestone in addressing an underserved therapeutic area, offering the first FDA-approved treatment specifically designed and clinically validated for fungal otitis externa in adult patients.

https://pubchem.ncbi.nlm.nih.gov/compound/Clotrimazole#section=2D-Structure

FDA Approves Clotic (clotrimazole otic solution), First Treatment with Clinical Evidence for Fungal Ear Infections

Bictegravir and lenacapavir: What is it and is it FDA approved?

2026-06-01T16:01:30.003+05:30

In continuation of my update on lenacapavir

lenacapavir

Bictegravir

Bictegravir plus lenacapavir (BIC/LEN) is an integrase strand transfer inhibitor (INSTI) and a capsid inhibitor combination in development for the treatment of HIV-1 infection.

Bictegravir was first FDA-approved as one of the ingredients in Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg) for the treatment of HIV-1 infection in 2018.

Lenacapavir was first FDA-approved under the brand name Sunlenca for treatment of HIV-1 infection 2022, and Yeztugo for pre‑exposure prophylaxis (PrEP) in 2025.

Bictegravir plus lenacapavir is a once-daily, single-tablet regimen designed to provide sustained virologic suppression with a high barrier to resistance.

A New Drug Application (NDA) has been submitted to the U.S. FDA for the treatment of HIV in adults who are virologically suppressed. The NDA submission was supported by positive Phase 3 data from the ARTISTRY-1 (NCT05502341) and ARTISTRY-2 (NCT06333808) trials, which evaluated BIC/LEN in adults with HIV with virological suppression, including those who switched from complex multi-tablet regimens or from Biktarvy. In both trials, BIC/LEN demonstrated comparable efficacy in maintaining virological suppression at Week 48 and was generally well tolerated, with no significant or new safety concerns identified.

- ARTISTRY-1 enrolled the oldest study population in a Phase 3 HIV treatment registrational trial to date. The Week 48 data presented at CROI 2026 showed the treatment switch to BIC/LEN from complex regimens was also associated with improvements in certain fasting lipid parameters and patient-reported treatment satisfaction. Detailed ARTISTRY-1 results were published in The Lancet on March 28, 2026.

- ARTISTRY-2 showed that switching to BIC/LEN had no significant impact on weight.

The FDA has granted priority review of the NDA and has assigned a Prescription Drug User Fee Act (PDUFA) action date of August 27, 2026

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

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

Bictegravir and lenacapavir: What is it and is it FDA approved?

Tomato-Soy Juice Shows Promise Against Chronic Inflammation

2026-05-30T14:08:45.871+05:30

A simple juice made from tomatoes and soybeans may help lower harmful inflammation linked with obesity and chronic disease, according to a new clinical study published in Molecular Nutrition & Food Research

Researchers from The Ohio State University and the United States Department of Agriculture found that adults with obesity who drank tomato-soy juice daily for four weeks showed reductions in several inflammation-related markers inside the body.

Scientists say the findings are important because chronic low-grade inflammation is increasingly linked with obesity, diabetes, heart disease, cancer, and other long-term illnesses.

Rather than focusing on supplements alone, researchers are now studying whether combinations of everyday foods may quietly influence inflammation and overall health over time.

Tomato-Soy Juice Shows Promise Against Chronic Inflammation

Could guava juice help prevent anemia?

2026-05-29T15:06:09.417+05:30

A systematic review from Indonesia, published in the journal BMJ Nutrition, Prevention & Health, indicates that adding guava juice to the diet could boost hemoglobin levels in adolescent girls and pregnant women. This could potentially offer a low-cost dietary complement to iron supplementation, given the high prevalence of iron-deficiency anemia among females, especially in low- and middle-income countries (LMICs).

Iron deficiency anemia among young women

In 2021, anemia was estimated to affect about 45 % of pregnant women and 39.5 % of non-pregnant women worldwide. Indonesia had similar figures: 48.9 % among pregnant women and 32 % among adolescent girls. Women with severe anemia are twice as likely to die during pregnancy and postpartum, compared to those with mild anemia.

Iron deficiency is the primary cause of anemia, especially in LMICs. The reasons include poor dietary intake, high prevalence of infection, heavy bleeding during periods, frequent pregnancies, and low healthcare access.

Iron deficiency anemia is conventionally treated with iron supplementation, but oral iron can cause symptoms such as diarrhea or constipation, other gut symptoms, an unpleasant taste, and may be inaccessible to some women. Pregnancy can further complicate treatment, as physiological changes may decrease iron absorption during this period.

This has resulted in persistently low use of iron supplements, even with national nutritional programs like the Gerakan Nasional Aksi Bergizi or iron supplementation programs targeting pregnant women and adolescent girls.

Nutritional benefits of guava

Guava is a locally cultivated and inexpensive fruit. Its juice is rich in vitamin C, folate, antioxidants, flavonoids, and polyphenols, and other micronutrients. The current study aimed to examine the potential of guava juice as a natural adjunct to iron therapy.

Guava juice and iron supplements

This systematic review and meta-analysis included 17 Indonesian studies published between 2019 and 2024, with a total of 726 participants. Most studies were quasi-experimental, while two were randomized controlled trials (RCTs). The participants were pregnant women or adolescent girls, with numbers ranging from 15 to 230.

While the findings were encouraging, most of the evidence came from relatively small quasi-experimental studies rather than randomized trials

Most studies evaluated guava juice alongside iron supplementation, while a few used it alone or in combination with carrot or red spinach juice. Intervention periods ranged from five days to three months.

Eight studies compared guava juice plus iron supplementation with iron supplementation alone, although only five of these provided extractable data for the direct comparative meta-analysis. One study used papaya juice and another dragon fruit juice as comparators. The remaining seven studies had no control group.

The meta-analysis was limited to 12 studies because the others lacked usable data. The results were promising, consistently indicating a significant average increase of 1.7 g/dL in hemoglobin among pooled participants who consumed guava juice.

When stratified by participant type, adolescents had a mean improvement in hemoglobin levels of 1.5 g/dL, versus 1.8 g/dL among pregnant women.

Across five studies that directly compared guava juice interventions with iron-only controls, hemoglobin levels increased by an additional 1.3 g/dL on average in the guava juice groups. This was confirmed to be robust by sensitivity analyses, with little evidence of publication bias.

Possible physiological pathways

The degree of improvement in hemoglobin observed by the researchers is substantial enough to potentially move some individuals with mild or moderate anemia into non-anemic categories. Other experimental studies involving male athletes, anemic schoolchildren, and postpartum women have shown similar positive effects from both guava juice and guava fruit consumption.

This suggests the beneficial impact of guava’s high vitamin C and polyphenol content, irrespective of the form of consumption. Vitamin C improves the absorption of iron from non-heme sources, including iron supplements, by converting ferric iron into its more absorbable ferrous form.

Guava also contains folate, antioxidants, flavonoids, and polyphenols that may support red blood cell survival by reducing oxidative stress. The researchers suggest that juice preparation may improve compliance and could enhance nutrient availability, while promoting more consistent intake.

Study limitations

Despite the promising results, the review also noted limitations. All studies were conducted in Indonesia, limiting generalizability to other populations. Many studies had a moderate risk of bias, sample sizes were relatively small, and most were non-randomized designs. Missing data also reduced the size of the meta-analysis. The studies showed high heterogeneity due to differences in guava dosage, preparation, and duration.

Future follow-up research should include larger, well-designed RCTs across multiple countries with standardized reporting, including regimens and outcomes. These should include not only hemoglobin but also parameters such as transferrin and ferritin that reflect broader health impacts and long-term effectiveness. This would help identify the optimal dosage, dosing frequency, and duration of use.

Implementation research is also required to understand how far such an intervention could be embedded into existing programs, supporting its real-world relevance.

Conclusion

Overall, the study concludes that guava juice significantly improves hemoglobin levels in women and adolescent girls. The authors highlight the potential of guava juice as an affordable, culturally acceptable, and locally available dietary adjunct to iron supplementation and anemia prevention programs in resource-limited settings.

It is particularly relevant in tropical countries with high guava production, such as India and Indonesia. The authors suggest integrating guava juice into school nutrition programs, antenatal care packages, and community health initiatives.

This could be a sustainable, complementary strategy to prevent and treat mild-to-moderate anemia, aligning with the United Nations’ Decade of Action on Nutrition (2016–2025) and its dietary emphasis on local foods.

Could guava juice help prevent anemia?

FDA Approves Rhapsido (remibrutinib) for the Treatment of Chronic Spontaneous Urticaria

2026-05-29T14:57:30.538+05:30

Novartis announced today that Rhapsido (remibrutinib) received US Food and Drug Administration (FDA) approval as an oral treatment for adult patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine treatment. Rhapsido is a pill taken twice daily and does not require injections or lab monitoring. It is the first FDA-approved Bruton's tyrosine kinase inhibitor (BTKi) for CSU. Rhapsido helps to inhibit the release of histamine and other proinflammatory mediators by targeting BTK, offering a unique approach to CSU treatment.

"CSU is a serious disease that can cause debilitating symptoms and unpredictable flares. It's difficult to diagnose and manage," said Mark Lebwohl, MD, Dean for Clinical Therapeutics at the Icahn School of Medicine at Mount Sinai and member of the steering committee for the remibrutinib REMIX Phase III clinical trial program. "Remibrutinib represents a new way of treating CSU. By blocking the activity of BTK, remibrutinib stops a key pathway of the immune response in CSU. This is an exciting new option that has the potential to help a broad range of patients get fast relief."

CSU is a mast cell-driven condition thought to be caused by immune dysregulation. In people with CSU, the immune system can become activated through allergic (IgE) or autoimmune (IgG) pathways.4 This causes certain immune cells—mast cells and basophils—to activate the BTK protein. While not fully understood, it is believed that once activated, BTK leads to the release of histamine and other proinflammatory mediators that may cause the red, swollen, and itchy hives commonly seen in CSU.5,6

CSU symptoms are unpredictable, recurring for six weeks or more without an identified cause.7 Diagnosis can take up to 24 months.8 Many CSU patients say their symptoms negatively impact their sleep, work, and mental health.9,10,11 Antihistamines are the first-line treatment, but over half of patients still have symptoms, even at higher doses.2 Injectable treatments exist for those who don't respond to antihistamines, yet fewer than 20% of eligible patients receive them.12

"The approval of remibrutinib is an important development in CSU care. It quickly reduces symptoms, offering patients control of the hives and itching that they experience on a daily basis," said Giselle Mosnaim, MD, MS, an Allergist and Immunologist from Endeavor Health, Clinical Associate Professor at the University of Chicago Pritzker School of Medicine and REMIX trial investigator. "This is significant because it expands beyond existing injectable treatments and gives patients an oral option that can easily be incorporated into their daily lives."

"Many CSU patients feel misunderstood and settle for treatments that don't fully meet their needs," said Lynda Mitchell, CEO of Allergy & Asthma Network. "We support new treatment options that empower patients to choose what works best for them. This convenient new oral therapy offers a promising new way to manage CSU and potentially improve daily life for those living with this challenging condition."

Clinical data supporting approval

The FDA approval of Rhapsido in CSU is based on results from the Phase III REMIX-1 (NCT05030311) and REMIX-2 (NCT05032157) clinical trials in patients who remained symptomatic on second-generation H1 antihistamines. Rhapsido demonstrated superiority in change from baseline versus placebo in itch (ISS7), hives (HSS7), and weekly urticaria activity (UAS7) at Week 12.13 Significantly more patients treated with Rhapsido versus placebo achieved well-controlled disease (UAS7≤6) as early as Week 2 and at Week 12, and about one-third of patients achieved complete absence of itch and hives at Week 12.13 Rhapsido has a demonstrated safety profile that requires no lab monitoring.13 The most common adverse events (incidence ≥3%) were nasal congestion, sore throat, and runny nose (nasopharyngitis), bleeding, headache, nausea, and abdominal pain.13

Novartis has completed regulatory submissions for Rhapsido for the treatment of CSU across many countries, including in the European Union, Japan, and China, with priority review granted in China.

Transforming care in Immunology

"This approval of Rhapsido as the first and only BTK inhibitor in CSU is an important milestone in our journey to reshape care for overlooked immune-related conditions and offer more patients the potential to find fast relief," said Victor Bultó, President, US, Novartis. "Building on our legacy in advancing the treatment of allergic, dermatologic, and rheumatologic conditions, we are deeply committed to further investing in innovative, patient-focused therapies across immunology."

Discovered and developed by Novartis to target the BTK pathway as a driver of inflammation, remibrutinib is being investigated in ongoing clinical trials across a variety of immune-related conditions, including chronic inducible urticaria (CIndU), hidradenitis suppurativa (HS), and food allergy.

Rhapsido (remibrutinib) Indication

Rhapsido is a prescription medicine used to treat adults with chronic spontaneous urticaria (CSU) who continue to have symptoms that are not controlled with antihistamine treatment. Rhapsido should not be used to treat any other forms of hives (urticaria). It is not known if Rhapsido is safe and effective in children.

Rhapsido Important Safety Information

Before taking Rhapsido, patients should tell their health care provider about all of their medical conditions, including if they:

Have had a recent surgery or plan to have surgery. Health care providers may tell patients to stop taking Rhapsido for at least 3 to 7 days before and after any planned medical or surgical procedures

Have bleeding problems or are taking a blood thinner medicine

Have liver problems

Are pregnant or plan to become pregnant. It is not known if Rhapsido will harm an unborn baby. If patients become pregnant during treatment with Rhapsido, they should talk to their health care provider about registering in the pregnancy exposure registry for Rhapsido

Are breastfeeding or plan to breastfeed. It is not known if Rhapsido passes into breast milk

Have recently received or are scheduled to receive an immunization (vaccine). Patients should talk to their health care provider about receiving any immunizations before taking Rhapsido. They should not receive live or live-attenuated vaccines during treatment with Rhapsido

Patients should tell their health care provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking Rhapsido with certain other medications may affect how Rhapsido or other medications work, and can cause side effects.

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

FDA Approves Rhapsido (remibrutinib) for the Treatment of Chronic Spontaneous Urticaria

FDA Approves Foundayo (orforglipron), the Only GLP-1 Pill for Weight Loss That Can be Taken Any Time of Day Without Food or Water Restrictions

2026-05-27T14:35:11.265+05:30

Eli Lilly and Company (NYSE: LLY) announced the FDA approval of Foundayo™ (orforglipron) for adults with obesity, or overweight with weight-related medical problems. When used alongside a reduced-calorie diet and increased physical activity, Foundayo helps individuals lose excess body weight and keep the weight off. Foundayo will be available via LillyDirect®, with prescriptions accepted immediately and shipping beginning April 6, followed shortly after by broad availability through U.S. retail pharmacies and telehealth providers.

Adults taking Foundayo lost an average of 27 pounds on the highest dose in the ATTAIN-1 clinical trial1

Foundayo, Lilly's second FDA-approved obesity medicine, will be available via LillyDirect® with free home delivery, starting at $25 per month with commercial coverage and $149 for self-pay

"People living with obesity need treatment options that meet them where they are – and for many, a once-daily pill that can be taken with no food or water restrictions can offer them greater flexibility in how they approach their treatment," said Deborah Horn, DO, director of the Center for Obesity Medicine at McGovern Medical School at UTHealth Houston. "With Foundayo, we now have an oral option that delivered an average of 12.4% weight loss at the highest dose in clinical trials – addressing both the clinical realities of obesity and the practical challenges patients face every day."

Supported by the rigorous ATTAIN clinical trial program, Foundayo was proven to help people lose weight and keep it off. In the ATTAIN-1 trial, individuals taking the highest dose of Foundayo and who stayed on treatment lost an average of 27.3 pounds (12.4%) compared to 2.2 pounds (0.9%) with placebo.1 Participants taking Foundayo, regardless of trial completion, lost an average of 25 pounds (11.1%), compared to 5.3 pounds (2.1%) with placebo.2 In the ATTAIN program, Foundayo also led to reductions in many markers of cardiovascular risk, including waist circumference, non-HDL cholesterol, triglycerides and systolic blood pressure across all doses.

"Today, fewer than 1 in 10 people who could benefit from a GLP-1 are taking one, held back by access, stigma, perceived complexity or the belief that their condition isn't serious enough for treatment. We believe Foundayo can help level the playing field for those living with obesity or who are overweight and living with weight-related complications," said David A. Ricks, chair and CEO of Eli Lilly and Company. "As a convenient, once-daily oral pill that delivers meaningful weight loss, this is obesity care designed for the real world."

Lilly is committed to making Foundayo accessible and affordable. Eligible people with commercial insurance may pay as little as $25 per month with the Foundayo savings card.3 Individuals opting for self-pay can access Foundayo starting at $149 per month for the lowest dose. Additionally, eligible Medicare Part D individuals may be able to get Foundayo for $50 per month, beginning as soon as July 1, 2026.

Use of Foundayo with other GLP-1 receptor agonist medicines is not recommended. It is not known whether Foundayo is safe and effective for use in children. Foundayo may cause tumors in the thyroid, including thyroid cancer. Watch for possible symptoms, such as a lump or swelling in the neck, hoarseness, trouble swallowing or shortness of breath. If you have any of these symptoms, tell your health care provider. The most common side effects of Foundayo include nausea, constipation, diarrhea, vomiting, indigestion, stomach (abdominal) pain, headache, swollen belly, feeling tired, belching, heartburn, gas, and hair loss. These are not all the possible side effects of Foundayo.

"There is no single path that works for everyone living with overweight or obesity," said Joe Nadglowski, president and CEO of the Obesity Action Coalition. "New treatment options expand choice and help more people find care that fits their lives, their goals and where they are in their journey – whether they're just starting to explore treatment or looking for a different long-term approach."

Lilly has submitted orforglipron for weight management and/or type 2 diabetes in more than 40 countries and plans to launch in each country shortly after approval

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

FDA Approves Foundayo (orforglipron), the Only GLP-1 Pill for Weight Loss That Can be Taken Any Time of Day Without Food or Water Restrictions

Vitamin C may reduce cancer-linked digestive chemical reactions

2026-05-25T14:23:25.690+05:30

In continuation of my update on Vitamin C

A new study from the University of Waterloo uses mathematical modeling to examine how Vitamin C affects chemical reactions in the digestive system that are linked to cancer development.

Over the last several decades, North American diets have seen a steady increase in exposure to nitrates and nitrites: compounds found in cured meats as well as fruits and vegetables grown using polluted soil and water. While nitrates and nitrites play important roles in neurological and heart health, in the stomach, they can undergo a chemical reaction known as "nitrosation" and form chemicals that many scientists suspect increase cancer risk.

Since at least the 90s, researchers have been studying the link between cancer and these compounds, with conflicting results. Our work suggests that the presence of dietary Vitamin C may help explain these inconsistencies."
Dr. Gordon McNicol, post-doctoral researcher in applied mathematics and first author of the study

The team built a mathematical model of the salivary glands, stomach, small intestine and plasma, and simulated how nitrites and nitrates move through the body and change over time. Their model demonstrated that when Vitamin C is also present in food, such as leafy greens like spinach, which contain both Vitamin C and nitrate, it could decrease cancer risk.

The study also suggested that taking Vitamin C supplements after each meal could have a moderate positive effect in reducing the formation of nitrosation products associated with cancer risk from dietary nitrites and nitrates, such as those found in foods like bacon and salami.

The researchers hope these findings will support future nutrition research.

"This work provides a mechanistic roadmap for future clinical and laboratory studies by identifying the key interacting drivers of these potentially harmful chemical reactions, including nitrite exposure, antioxidant intake, meal timing, gastric conditions and oral microbiome activity," said Dr. Anita Layton, professor of applied mathematics and Canada 150 researcher chair. "This model can help researchers design more targeted experiments and interventions, focusing on when and in whom nitrosation is most likely to occur."

The research, "Vitamin C as a nitrosation inhibitor: A modelling study across dietary patterns and water quality," appears in the Journal of Theoretical Biology.

https://www.sciencedirect.com/science/article/pii/S002251932600069X?via%3Dihub

Vitamin C may reduce cancer-linked digestive chemical reactions

FDA Approves Lasix ONYU (furosemide) for Treatment of Edema in Heart Failure

2026-05-21T09:22:58.627+05:30

SQ Innovation, Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved its drug-device combination Lasix ONYU (furosemide injection) for the treatment of edema (due to fluid overload) in adult patients with chronic heart failure. Lasix ONYU was developed to enable subcutaneous infusion of furosemide outside the healthcare setting for selected patients, as prescribed by a clinician without the need for a healthcare professional to administer the drug.

About 6.7 million Americans suffer from heart failure, with the prevalence expected to rise to 8.7 million by 2030. Heart failure is a leading cause of hospitalizations for individuals aged 65 and older with approximately 1.2 million hospitalizations per year [1].

Lasix ONYU consists of a novel high-concentration formulation of furosemide combined with a state-of-the-art small Infusor for treatment at home. The innovative design includes a reusable unit that can be used for 48 treatments and a plastic sterile single-use unit that is discarded after treatment. The two-component design reduces manufacturing complexity and cost, allowing the product to be offered at a different, more favorable price point which is expected to reduce barriers to widespread adoption.

“Lasix ONYU has the potential to be transformative in the care of patients experiencing worsening heart failure due to fluid overload,” said Pieter Muntendam, MD, founder, President and CEO of SQ Innovation. “Treating selected patients at home offers important benefits to patients, health systems and payors. We look forward to launching Lasix ONYU with leading health systems in the 4th quarter of 2025.”

Bioavailability and diuretic response were determined in a clinical study in which Lasix ONYU demonstrated complete bioavailability (112%) resulting in similar diuresis (115%) and natriuresis (117%) when compared to the same dose given by IV bolus. The biphasic delivery of furosemide by the Infusor resulted in a tempered diuretic response while IV bolus administration led to a shorter period of more intense diuresis. The results of the study were published in a leading cardiovascular journal [2].

“Heart failure is the most common serious medical condition in the U.S. and affects about one in four Americans during their lifetime. The number of patients affected is expected to double over the next 20 years and we currently already often lack adequate resources to take care of the 6.7 million patients affected presently – there are not enough beds, clinicians and funds”, said Dr. Javed Butler, Professor of Medicine at University of Mississippi and President, Baylor Scott and White Research Institute. “The only two actionable solutions now are more widespread adoption of guideline directed medical therapy (GDMT) and treating more patients at home with products such as subcutaneous diuretics instead of hospitalization for intravenous diuretics.”

“Decongestion through use of IV diuretics has been the cornerstone of treatment for reducing edema and hypervolemia in heart failure patients for over five decades,” stated S. Craig Thomas, Immediate Past President of the American Association of Heart Failure Nurses (AAHFN), an organization dedicated to advancing nursing education, clinical practice, and research to improve outcomes for heart failure patients. “The availability of accessible, affordable, and novel options that do not require the presence of a healthcare professional allows for transformative new clinical care-delivery. This means patients who now would typically need to be hospitalized for several days of IV treatment can instead remain home, supported by periodic or remote monitoring. The significance of this shift away from inpatient care for patients, hospitals, and payers cannot be overstated.”

Starting this quarter, Lasix ONYU will be available from leading pharmaceutical distributors enabling timely availability at participating medical facilities and affiliated retail pharmacies.

SQ Innovation is hosting a Conference Call and Webcast on Thursday October 9, at 4:30pm ET to introduce the product and answer questions from the community. Participating in the conference call will be:

Pieter Muntendam, MD, President and CEO of SQ Innovation

Mustafa M. Ahmed, MD, Professor of Medicine, Section Chief, Heart Failure, University of Florida Health, Gainesville, FL

Craig Thomas, Nurse Practitioner, Advanced Heart Failure Center, University of Virginia Health System, Charlottesville, VA and Immediate Past President American Association of Heart Failure Nurses (AAHFN)

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

FDA Approves Lasix ONYU (furosemide) for Treatment of Edema in Heart Failure

FDA Approves Jascayd (nerandomilast) to Treat Idiopathic Pulmonary Fibrosis

2026-05-20T13:46:56.312+05:30

Boehringer Ingelheim’s Jascayd (nerandomilast) tablets has been approved by the U.S. Food and Drug Administration (FDA) as an oral treatment option for idiopathic pulmonary fibrosis (IPF) in adult patients.2 Jascayd is the first and only preferential inhibitor of phosphodiesterase 4B (PDE4B) to be approved in this indication. This represents a novel mechanism of action that exerts both antifibrotic and immunomodulatory effects, thereby slowing the decline in lung function in IPF patients.2,3

“This milestone represents a new era in the treatment of IPF, a rare and debilitating chronic condition that worsens lung function. Nerandomilast has proven to slow lung function decline in IPF,” said Toby Maher, M.D., Ph.D., Professor of Clinical Medicine, Keck School of Medicine, USC Los Angeles. “Nerandomilast is a welcome new treatment option with a well-tolerated safety profile for physicians to consider for appropriate patients.”

The FDA approval is based on data from two clinical trials: FIBRONEER™-IPF (NCT05321069) and Trial 2 (NCT04419506). The primary endpoint in FIBRONEER™-IPF was the absolute change from baseline in Forced Vital Capacity (FVC), a measure of lung function,4 in mL at week 52.2,3 Nerandomilast demonstrated a significantly smaller FVC decline compared to placebo.2,3 Specifically, the adjusted mean decline in patients receiving 18 mg or 9 mg nerandomilast was -106 mL and -122 mL, respectively, versus -170 mL in the placebo group.2 Additionally, a treatment effect was shown as early as week two with nerandomilast 18 mg compared to placebo, with changes from baseline in FVC continuing to diverge over time to week 52.2,3

“The FDA approval of nerandomilast is a pivotal moment for people living with IPF as this marks the first time in over 10 years that the treatment landscape is evolving,” said Shashank Deshpande, Chairman of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “This new step forward, driven by the compelling results of the FIBRONEER™-IPF trial, underscores our unwavering commitment to change the way we treat IPF by delivering innovative therapies like nerandomilast.”

The most common (≥5%) adverse reactions reported in patients treated with nerandomilast and more frequently than the placebo group were as follows for nerandomilast 18 mg, 9 mg and placebo, respectively: diarrhea (42%, 31%, 17%), COVID-19 (13%, 16%, 12%), upper respiratory tract infection (13%, 11%, 10%), depression (12%, 11%, 10%), weight decreased (11%, 10%, 8%), decreased appetite (9%, 9%, 5%), nausea (8%, 9%, 7%), fatigue (7%, 8%, 6%), headache (7%, 6%, 5%), vomiting (6%, 5%, 5%), back pain (6%, 5%, 4%) and dizziness (5%, 6%, 5%).2

Discontinuation due to adverse reactions occurred more frequently in patients treated with nerandomilast (with or without background antifibrotic treatment) 18 mg (15%) and 9 mg (12%) compared to placebo (11%).2 The most frequent adverse reaction leading to discontinuation of nerandomilast 18 mg and 9 mg was diarrhea (6% and 2%, respectively).2

There is no ‘Warnings and Precautions’ section in the FDA approved product label.2

“The FDA approval of nerandomilast is exciting news for people living with idiopathic pulmonary fibrosis and their caregivers,” said Scott Staszak, President and CEO of the Pulmonary Fibrosis Foundation. “There has been a long-standing need for new treatment options for IPF within our community and nerandomilast provides an important addition to the care landscape.”

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

FDA Approves Jascayd (nerandomilast) to Treat Idiopathic Pulmonary Fibrosis

Mavacamten improves obstruction in adolescents with hypertrophic cardiomyopathy

2026-05-19T11:23:11.454+05:30

In continuation of my update on Mavacamten

Adolescent patients with obstructive hypertrophic cardiomyopathy (HCM) who received the drug mavacamten saw a significant improvement in left ventricular outflow tract (LVOT) gradient, a measure of blood flow obstruction in the heart, compared with those who received a placebo, according to a small study presented at the American College of Cardiology's Annual Scientific Session (ACC.26). The trial is the first to test mavacamten in patients younger than 18. This study was simultaneously published online in the New England Journal of Medicine at the time of presentation.

"These results are very encouraging," said Joseph William Rossano, MD, chief of cardiology at Children's Hospital of Philadelphia and the study's lead author. "Patients feel better, and their hearts look better."

HCM is a genetic disorder in which the heart muscle thickens, causing the heart chambers to become smaller and potentially reducing its ability to pump blood. In many cases, the thickened muscle blocks or reduces the flow of blood into the aorta from the left ventricle, known as obstructive HCM. Obstructive HCM can cause symptoms such as chest pain, dizziness, shortness of breath and swelling and lead to heart failure and death.

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

Mavacamten improves obstruction in adolescents with hypertrophic cardiomyopathy

Investigational drug delivers mixed results for uncontrolled blood pressure

2026-05-18T15:34:15.417+05:30

The investigational drug tonlamarsen—which is designed to lower blood pressure by reducing the production of angiotensinogen, a protein that turns into the hormone that regulates blood pressure—caused a significant and sustained drop in angiotensinogen but its impact on blood pressure was less clear, according to a study presented.

https://searchusan.ama-assn.org/usan/documentDownload?uri=/unstructured/binary/usan/tonlamarsen-sodium.pdf

Investigational drug delivers mixed results for uncontrolled blood pressure

Probing a paradoxical drug response for irregular heartbeat

2026-05-15T10:50:12.925+05:30

Mexiletine

https://go.drugbank.com/drugs/DB00379

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

Irregular heartbeat, or arrhythmia, can be treated with various procedures or medication, but not all medications work for all patients. In fact, one arrhythmia medication can actually cause arrhythmia in people with a common genetic variant. This problem creates a need for personalized medicine to provide patients with the most beneficial outcome, according to new research from WashU

Jon Silva, a professor of biomedical engineering in the McKelvey School of Engineering at Washington University in St. Louis, and Martina Marras, a doctoral student in his lab, made their discovery by screening four genetic variants to see if any of them altered the effectiveness of the drug mexiletine. The work is published in The Journal of Precision Medicine: Health and Disease.

Probing a paradoxical drug response for irregular heartbeat

https://medicalxpress.com/news/2026-01-stem-cell-expert-qa-pathways.html?utm_source=embeddings&utm_medium=related&utm_campaign=internal

FDA Approves Ferabright (ferumoxytol injection) Contrast Agent for Magnetic Resonance Imaging of the Brain

2026-05-14T15:04:46.257+05:30

Azurity Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) approval of Ferabright (ferumoxytol injection), the first and only iron-based contrast agent indicated for magnetic resonance imaging (MRI) of the brain in adults with known or suspected malignant neoplasms in the brain to visualize lesions with a disrupted blood-brain barrier.1

“Ferabright is an advanced superparamagnetic iron oxide nanoparticle contrast agent engineered for high relaxivity,” said Ronald Scarboro, CEO of Azurity Pharmaceuticals. “It significantly enhances image contrast and precision in brain tumor delineation compared to non-contrast MRI, offering clinicians a new tool for diagnostic imaging and improved patient care.”

In a clinical trial, Ferabright-enhanced MRI significantly improved visualization of primary and secondary brain neoplasms compared with pre-contrast imaging.1 In addition to improved imaging, Ferabright offers an extended imaging window due to its long half-life, which may support flexible MRI protocols without the need for multiple contrast administrations.1 As an iron-based agent, Ferabright is processed through the body’s natural iron metabolism pathways,3,6,7 potentially reducing concerns related to long-term retention associated with other contrast agents.2,8 And unlike many other agents, Ferabright is suitable for patients with renal insufficiency,2,3,4 enabling access to contrast-enhanced MRI while reducing exposure to heavy metals found in other contrast agents.5

“Ferumoxytol expands our MRI toolkit and provides an option for patients who are either contraindicated for or decline gadolinium,” said Csanad Varallyay, MD, PhD, Neuroradiologist based in Portland, Oregon, with longstanding research experience in ferumoxytol-enhanced MRI.

Ferabright will be supplied in single-dose vials of 300 mg elemental iron per 10 mL (30 mg/mL) and 510 mg elemental iron per 17 mL (30 mg/mL) for intravenous infusion over at least 15 minutes.1 Ferabright is contraindicated in patients with known hypersensitivity to ferumoxytol, any of Ferabright’s components, or any other intravenous iron products. Reactions have included anaphylaxis.1

https://www.chemicalbook.com/ChemicalProductProperty_EN_CB92493638.htm

FDA Approves Ferabright (ferumoxytol injection) Contrast Agent for Magnetic Resonance Imaging of the Brain

Small molecule could slow or stop progress of Parkinson's disease and related brain disorders, not just treat symptoms

2026-05-11T11:30:07.249+05:30

A team of researchers from NYU Abu Dhabi and the University of Denver has identified a promising small molecule that could help slow or halt the progression of serious brain diseases such as Parkinson's disease, offering new hope for treatments that go beyond managing symptoms. These diseases, which also include Lewy body dementia and multiple system atrophy, are caused by aggregation and spread of a neuronal protein, which damages brain cells over time. Currently, there are no approved treatments that can stop or slow this process.

https://medicalxpress.com/news/2026-03-small-molecule-parkinson-disease-brain.html

Small molecule could slow or stop progress of Parkinson's disease and related brain disorders, not just treat symptoms

Unicycive Therapeutics Announces Receipt of Complete Response Letter for Oxylanthanum Carbonate for the Treatment of Hyperphosphatemia in Patients with Chronic Kidney Disease on Dialysis

2026-05-08T10:42:11.850+05:30

Unicycive Therapeutics, Inc. (“Unicycive” or the “Company”) (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease, announced the U.S. Food and Drug Administration (FDA) issuing a CRL for its New Drug Application (NDA) for OLC to treat hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis.

“We plan to immediately seek a Type A meeting with the Agency to gain alignment on the best strategy to ensure rapid resolution of the CRL,” said Shalabh Gupta, M.D., Chief Executive Officer of Unicycive. “With a second manufacturing vendor identified that has produced OLC drug product, we remain optimistic about our ability to bring this promising new treatment option to patients with CKD on dialysis who are managing hyperphosphatemia, and we plan to provide an update as soon as we have additional clarity on next steps from the FDA.”

After submitting the NDA, and as a part of the application review and routine information requests, the FDA notified Unicycive that a third-party manufacturing vendor of its main contract development and manufacturing organization (CDMO) was cited for deficiencies following a cGMP inspection. This citation is unrelated to OLC. Unicycive also notes that as part of the NDA review, the Agency has not highlighted any other technical concerns related to the submitted CMC documentation or testing of OLC itself.

As part of its overall manufacturing strategy, the Company had previously identified a back-up third-party manufacturing vendor to build redundancy into its supply chain. The second vendor has a long history of successful FDA and international regulatory inspections and has already produced OLC drug product, which could also be used to support the resolution of the CMC issues identified in the CRL.

About Oxylanthanum Carbonate (OLC)

OLC is an investigational oral phosphate binder that leverages proprietary nanoparticle technology to deliver high phosphate binding potency, reducing the number and size of pills that patients must take to treat hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis. Its potential best-in-class profile may have meaningful patient adherence benefits over currently available treatment options as it requires a lower pill burden.

Unicycive is seeking FDA approval of OLC via the 505(b)(2) regulatory pathway. The NDA submission package is based on data from three clinical studies (a Phase 1 study in healthy volunteers, a bioequivalence study in healthy volunteers, and a tolerability study of OLC in CKD patients on dialysis), multiple preclinical studies, and the chemistry, manufacturing and controls (CMC) data. OLC is protected by a strong global patent portfolio including issued patents on composition of matter with exclusivity until 2031, and with the potential for patent term extension until 2035.

About Hyperphosphatemia

Hyperphosphatemia is a serious medical condition that occurs in nearly all patients with End Stage Renal Disease (ESRD). Annually there are over 450,000 individuals in the U.S. that require medication to control their phosphate levels.1 Uncontrolled hyperphosphatemia is strongly associated with increased death and hospitalization for CKD patients on dialysis. Treatment of hyperphosphatemia is aimed at lowering serum phosphate levels via two means: (1) restricting dietary phosphorus intake; and (2) using, on a daily basis, and with each meal, oral phosphate binding drugs that facilitate fecal elimination of dietary phosphate rather than its absorption from the gastrointestinal tract into the bloodstream.

Unicycive Therapeutics Announces Receipt of Complete Response Letter for Oxylanthanum Carbonate for the Treatment of Hyperphosphatemia in Patients with Chronic Kidney Disease on Dialysis

Aldeyra Therapeutics Announces FDA Acceptance for Review of Reproxalap New Drug Application for the Treatment of Dry Eye Disease

2026-05-07T11:15:00.001+05:30

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) announced the U.S. Food and Drug Administration (FDA) acceptance the review of the resubmitted New Drug Application (NDA) for topical ocular reproxalap, a first-in-class investigational new drug candidate, for the treatment of the signs and symptoms of dry eye disease. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 16, 2025.

“Based on the FDA’s requirement for an additional clinical trial demonstrating the efficacy of reproxalap in treating the symptoms of dry eye disease, and per agreement with the FDA, the NDA resubmission contained a single clinical trial that achieved the primary endpoint of reducing ocular discomfort relative to the vehicle control,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra. “We look forward to a productive dialog with the FDA during the NDA review of reproxalap, which, to our knowledge, remains the only dry eye disease investigational therapy to have demonstrated acute activity in reducing ocular discomfort and redness in pivotal trials simulating the disease flares that are likely the most bothersome aspects of dry eye disease.”

About Reproxalap

Reproxalap is an investigational new drug candidate in development for the treatment of dry eye disease and allergic conjunctivitis, two of the largest markets in ophthalmology. Reproxalap is a first-in-class small-molecule modulator of RASP, which are elevated in ocular and systemic inflammatory diseases. The mechanism of action of reproxalap has been supported by the demonstration of statistically significant and clinically relevant activity in multiple physiologically distinct late-phase clinical indications. Reproxalap has been studied in more than 2,900 patients with no observed safety concerns; mild and transient instillation site irritation is the most commonly reported adverse event in clinical trials.

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

Aldeyra Therapeutics Announces FDA Acceptance for Review of Reproxalap New Drug Application for the Treatment of Dry Eye Disease

FDA grants speedy approval to Eli Lilly's weight-loss pill for obesity

2026-04-30T09:54:02.167+05:30

Federal regulators on Wednesday approved Eli Lilly's new weight-loss pill, a second daily oral medication to treat obesity and other weight-related conditions.

The Food and Drug Administration granted expedited approval to orforglipron, a GLP-1 drug that works like widely used injectable medications to mimic a natural hormone that controls appetite and feelings of fullness.

The drug, which will be branded as Foundayo, is expected to begin shipping Monday. The company said people with insurance may be able to get the drug starting at $25 per month with a Lilly discount card. Prices for people paying cash will range between $149 per month to $349 per month, depending on the dose.

The new pill joins drugmaker Novo Nordisk's oral Wegovy pill, which has spurred more than 600,000 prescriptions in the United States since it was approved in December.

The FDA authorized Eli Lilly's drug as part of a new program aimed at cutting drug approval times. The agency said it reviewed the company's application in 50 days.

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FDA grants speedy approval to Eli Lilly's weight-loss pill for obesity

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

Study Details | NCT07506044 | Dexmedetomidine as an Adjuvant During Dural Puncture Epidural

2026-04-29T09:30:59.950+05:30

In continuation of my update on Dexmedetomidine

Study Overview

Brief Summary:

The study aimed to determine the effects of using varied doses of DEX on the concentration of bupivacaine injected for epidural labor analgesia, and to assess the benefits of the dural puncture epidural.

Detailed Description

The primary efficacy point is the total dose of bupivacaine administered during labor. The secondary endpoints include the VAS pain scores, number of administered additional doses, incidence of motor block, delivery type (spontaneous vaginal or cesarean), maternal satisfaction, and complications such as hypotension, fetal bradycardia, and epidural-related maternal fever.

Official Title

The Effect of Different Doses of Dexmedetomidine as an Adjuvant to Bupivacaine for Parturients Having Dural Puncture Epidural Technique to Elaborate Labor Pain.

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

Study Details | NCT07506044 | Dexmedetomidine as an Adjuvant During Dural Puncture Epidural | ClinicalTrials.gov