Med-Chemist

Daily coffee drinking may slow biological aging of people with major mental illness

2026-04-24T14:06:00.001+05:30

Drinking a maximum of 3–4 cups of coffee a day may slow the "biological" aging of people with severe mental illness, by lengthening their telomeres—indicators of cellular aging—and giving them the equivalent of 5 extra biological years, compared with non-coffee drinkers, finds research published in BMJ Mental Health.

But no such effects were observed beyond this quota, which is the maximum daily intake recommended by several international health authorities, including the NHS and the US Food and Drug Administration.

Telomeres sit at the end of chromosomes and perform a role similar to the plastic tips on the end of shoelaces. While telomere shortening is a natural part of the aging process, it seems to be accelerated in those with major psychiatric disorders, such as psychosis, schizophrenia, and bipolar disorder, note the researchers.

Study details and participant information

Telomeres are sensitive to environmental factors, including, possibly, diet. And coffee, when drunk in moderation, has been associated with various health benefits, prompting the researchers to explore whether it might influence the rate at which telomeres shorten in people with major mental ill health.

They included 436 adult participants from the Norwegian Thematically Organized Psychosis (TOP) study, recruited between 2007 and 2018: 259 had schizophrenia; the rest (177) had affective disorders, including bipolar disorder and major depressive disorder with psychosis.

Participants were asked how much coffee they drank every day and were grouped into four categories: zero (44); 1–2 cups; 3–4 cups (110); and 5 or more cups. And they were asked whether they smoked, and if so, for how long they had done so.

Participants who drank 5+ cups a day were significantly older than those who drank none or 1–2 cups a day. And those with schizophrenia drank significantly more coffee than those with an affective disorder.

Coffee intake is associated with telomere length in severe mental disorders, BMJ Mental Health (2025). DOI: 10.1136/bmjment-2025-301700

Daily coffee drinking may slow biological aging of people with major mental illness

Johnson & Johnson Seeks First Icotrokinra U.S. FDA Approval Aiming to Revolutionize Treatment Paradigm for Adults and Adolescents with Plaque Psoriasis

2026-04-22T13:55:00.004+05:30

Johnson & Johnson (NYSE: JNJ) today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking the first approval of icotrokinra, a first-in-class investigational targeted oral peptide that selectively blocks the IL-23 receptor for the treatment of adults and pediatric patients 12 years of age and older with moderate to severe plaque psoriasis (PsO). Icotrokinra is uniquely designed to block the IL-23 receptor, which underpins the inflammatory response in plaque PsO and offers potential in other IL-23-mediated diseases.1,2,3

The application included data from four pivotal Phase 3 studies conducted as part of the ICONIC clinical development program, including ICONIC-LEADa, ICONIC-TOTALb and ICONIC-ADVANCE 1 & ICONIC-ADVANCE 2c. Treatment with icotrokinra met all primary and co-primary endpoints across the development program among adults and pediatric patients 12 years of age and older with moderate-to-severe plaque PsO, demonstrating significant skin clearance and a favorable safety profile in a once-daily pill. Results from the ICONIC-ADVANCE 1 & 2 studies show icotrokinra achieved co-primary endpoints and showed superiority to deucravacitinib in moderate-to-severe plaque PsO. Across all studies, pooled safety data showed a similar proportion of patients experienced adverse events (AEs) between icotrokinra (49.1%) and placebo (51.9%) groups, with no new safety signals identified to date.4,5,6,7,8

“The rapid patient enrollment across our ICONIC clinical program underscores the unmet need for an advanced plaque psoriasis treatment that meaningfully addresses their needs and preferences,” said Liza O’Dowd, MD, Vice President, Immunodermatology and Respiratory Disease Area Lead, Johnson & Johnson Innovative Medicine. “Given the breadth and depth of our studies, along with the robust clinical results reported to date, we are confident that icotrokinra has the potential to transform how physicians and patients think about plaque psoriasis care, establishing a new standard in the treatment of this immune-mediated disease.”

Data submitted to the FDA as part of the NDA include:

Results from the Phase 3 ICONIC-LEAD study, presented as a late-breaking abstract at the 2025 American Academy of Dermatology (AAD) Annual Meeting, that showed icotrokinra successfully met the co-primary endpoints of Investigator’s Global Assessment (IGA)d score of 0/1 (clear or almost clear skin) and Psoriasis Area and Severity Index (PASI)e 90 compared to placebo at Week 16.4
A subgroup analysis of ICONIC-LEAD, presented at the 2025 World Congress of Pediatric Dermatology (WCPD), which demonstrated pediatric patients 12 years of age and older treated with once daily icotrokinra achieved higher rates of clear or almost clear skin at Week 16 compared to patients receiving placebo with no new safety signals identified.5
Data from the Phase 3 ICONIC-TOTAL study, presented at the 2025 Society for Investigative Dermatology (SID) Annual Meeting, that highlighted the potential of icotrokinra in patients with difficult-to-treat scalp and genital psoriasis.6
Results from the Phase 3 ICONIC-ADVANCE 1 & ICONIC-ADVANCE 2 studies, that further reinforced the overall efficacy profile met co-primary endpoints of IGA 0/1 and PASI 90 versus placebo at Week 16. Icotrokinra also met all key secondary endpoints at Weeks 16 and 24 that measured superiority to deucravacitinib in patients with moderate-to-severe plaque PsO.7,8 Comprehensive results are being prepared for presentation at a future medical meeting.
Long-term data from the ICONIC development program, including at least 52-weeks of treatment for ICONIC-LEAD and ICONIC-TOTAL, and results from a randomized withdrawal analysis evaluating the durability of response, are being prepared for presentation at a future medical meeting.

Johnson & Johnson has also initiated the Phase 3 ICONIC-ASCENDf study, the first-ever head-to-head study seeking to demonstrate the superiority of an oral pill, icotrokinra, compared to an injectable biologic, ustekinumab, representing an important step forward in psoriasis research.9

ICONIC-LEAD is a Phase 3 randomized controlled trial (RCT) evaluating the efficacy and safety of icotrokinra compared with placebo in 684 participants (icotrokinra=456; placebo=228) 12 years of age or older with moderate-to-severe plaque PsO, with the higher efficacy bar of PASI 90 and IGA score of 0/1 with at least a 2-grade improvement as co-primary endpoints. ICONIC-LEAD enrolled 66 adolescent patients.
ICONIC-TOTAL is a Phase 3 RCT evaluating the efficacy and safety of icotrokinra compared with placebo for the treatment of plaque PsO in 311 participants (icotrokinra=208; placebo=103) with at least moderate severity affecting special areas (e.g., scalp, genital and/or hands and feet) with overall IGA score of 0 or 1 with at least a 2-grade improvement as the primary endpoint.
ICONIC- ADVANCE 1 & 2 are Phase 3 RCTs evaluating the efficacy and safety of icotrokinra compared with placebo and deucravacitinib in participants with moderate-to-severe plaque PsO with PASI 90 and IGA score of 0/1 with at least a 2-grade improvement as co-primary endpoints.
The IGA is a five-point scale with a severity score ranging from 0 to 4, where 0 indicates clear, 1 is minimal, 2 is mild, 3 is moderate and 4 indicates severe disease.10
The PASI score grades the amount of surface area on each body region that is covered by psoriasis plaques and the severity of plaques for their redness, thickness and scaliness.11 PASI 90 corresponds to an improvement of >=90% in PASI score from baseline.11
ICONIC-ASCEND is a Phase 3 RCT and the first-ever head-to-head study seeking to demonstrate the superiority of an oral pill, icotrokinra, compared to an injectable biologic, ustekinumab in moderate-to-severe plaque PsO

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

Johnson & Johnson Seeks First Icotrokinra U.S. FDA Approval Aiming to Revolutionize Treatment Paradigm for Adults and Adolescents with Plaque Psoriasis

ACC: Mavacamten Efficacious for Adolescents With Obstructive Hypertrophic Cardiomyopathy

2026-04-16T09:47:00.005+05:30

In continuation of my update on Mavacamten

Mavacamten, the first-in-class cardiac myosin inhibitor approved for treatment of symptomatic adults with obstructive hypertrophic cardiomyopathy (HCM), is efficacious for adolescents with obstructive HCM, according to a study published online March 29 in the New England Journal of Medicine to coincide with the annual meeting of the American College of Cardiology, held from March 28 to 30 in New Orleans.

Joseph W. Rossano, M.D., from the Children's Hospital of Philadelphia, and colleagues conducted a randomized international study assessing the efficacy and safety of mavacamten in adolescents (aged 12 to <18 years) with obstructive HCM. After five weeks of screening, 44 participants were randomly assigned to either mavacamten (2.5 or 5 mg/day based on body weight) or placebo for 28 weeks.

The researchers found that participants receiving mavacamten had a substantial improvement in terms of the change in Valsalva left ventricular outflow tract (LVOT) gradient from baseline to week 28, with average decreases of 48.5 and 0.5 mmHg in the mavacamten and placebo groups, respectively. Significant improvements in favor of mavacamten were also seen in secondary end points, including change in resting LVOT gradient, maximal left ventricular wall thickness, peak oxygen consumption, and symptom measures such as fatigue and shortness of breath. Troponin and peptide levels decreased in those taking mavacamten and increased in those taking placebo.

"Beyond symptom relief, there's a signal that this may be favorably remodeling the heart, which could improve the natural history of the disease," Rossano said in a statement. "This suggests that it could be important to start children on this therapy when they’re young, before they've had many decades of ongoing injury to the heart from the obstruction."

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

ACC: Mavacamten Efficacious for Adolescents With Obstructive Hypertrophic Cardiomyopathy - Drugs.com MedNews

AAD: Significant Benefits Seen for Brepocitinib 30 mg in Dermatomyositis -

2026-04-15T09:46:00.002+05:30

For adults with dermatomyositis, brepocitinib, an oral tyrosine kinase 2/Janus kinase 1 inhibitor, demonstrates significant benefits at a 30-mg dose compared with placebo, according to a study published online March 28 in the New England Journal of Medicine to coincide with the annual meeting of the American Academy of Dermatology, held from March 27 to 31 in Denver.

Ruth Ann Vleugels, M.D., M.P.H., from Harvard Medical School in Boston, and colleagues conducted a phase 3, double-blind, randomized trial involving adults with dermatomyositis. Participants were randomly assigned to oral brepocitinib 30 mg, brepocitinib 15 mg, or placebo once daily for 52 weeks (81, 81, and 79 participants, respectively).

The researchers found that the mean Total Improvement Scores (TIS) at week 52 were 46.5, 37.5, and 31.2, respectively (brepocitinib 30 mg versus placebo difference: 15.3). On all nine key secondary end points, brepocitinib 30 mg was superior to placebo, including Cutaneous Dermatomyositis Disease Severity Index-Activity at week 4, moderate TIS response at week 52 with ≤2.5 mg/day of prednisone equivalent, and Health Assessment Questionnaire-Disability Index at week 52. The brepocitinib 30-mg arm had an increase in serious infections (9.9 percent versus 1.3 percent in placebo), but malignancies, thromboembolic events, and cardiovascular events were more frequent with placebo. There were no deaths reported.

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

AAD: Significant Benefits Seen for Brepocitinib 30 mg in Dermatomyositis

Arvinas Announces FDA Acceptance of the New Drug Application for Vepdegestrant for the Treatment of ESR1m, ER+/HER2- Advanced Breast Cancer

2026-04-11T09:47:00.004+05:30

Arvinas, Inc. (Nasdaq: ARVN), today with its partner Pfizer Inc. (NYSE: PFE), announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vepdegestrant for the treatment of patients with estrogen receptor-positive (ER+)/human epidermal growth factor receptor 2-negative (HER2-), ESR1-mutated advanced or metastatic breast cancer who have previously received endocrine-based therapy. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of June 5, 2026.

“Patients often face limited treatment options after first-line treatment and vepdegestrant demonstrated improved progression-free survival in patients with ESR1-mutated ER+/HER2- advanced breast cancer,” said John Houston, Ph.D., Chairperson, Chief Executive Officer, and President at Arvinas. “With the efficacy and favorable tolerability seen in VERITAC-2, we believe vepdegestrant, if approved, has potential to be a best-in-class treatment option for patients in the second-line ESR1-mutant setting. We look forward to working alongside Pfizer and with the FDA to pursue vepdegestrant’s approval and to ensure this important treatment option is made available to patients as rapidly as possible.”

Vepdegestrant, an investigational oral PROTAC ER degrader, is being jointly developed by Arvinas and Pfizer. The NDA submission was based on data from VERITAC-2 (NCT05654623), a global, randomized Phase 3 clinical trial evaluating vepdegestrant versus fulvestrant. These data were recently presented at the American Society of Clinical Oncology (ASCO) 2025 Annual Meeting and were simultaneously published in The New England Journal of Medicine.

About Vepdegestrant

Vepdegestrant is an investigational, orally bioavailable PROteolysis TArgeting Chimera (PROTAC) estrogen receptor degrader. Vepdegestrant is being developed as a potential monotherapy for ER+/HER2- advanced or metastatic breast cancer with estrogen receptor 1 (ESR1) mutations in the second line-plus setting.

In July 2021, Arvinas announced a global collaboration with Pfizer for the co-development and co-commercialization of vepdegestrant; Arvinas and Pfizer will share worldwide development costs, commercialization expenses, and profits.

The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vepdegestrant for its use as a monotherapy in the treatment of adults with estrogen receptor–positive (ER+), human epidermal growth factor receptor 2–negative (HER2-), ESR1-mutated advanced or metastatic breast cancer previously treated with endocrine-based therapy. Vepdegestrant has also been granted Fast Track designation by the FDA, underscoring the significant unmet need in this patient population and the potential for vepdegestrant to offer a meaningful new treatment option.

About the VERITAC-2 Clinical Trial

The Phase 3 VERITAC-2 clinical trial (NCT05654623) is a global, randomized trial evaluating the efficacy and safety of vepdegestrant (ARV-471) as a monotherapy compared to fulvestrant in patients with ER+/HER2- advanced or metastatic breast cancer previously treated with a CDK4/6 inhibitor plus endocrine therapy. The trial enrolled 624 patients, 270 of whom had ESR1m positive disease, at 213 sites in 25 countries.

Patients were randomized 1:1 to receive either vepdegestrant once daily, orally on a 28-day continuous dosing schedule, or fulvestrant, administered intramuscularly on Days 1 and 15 of Cycle 1 and then on Day 1 of each 28-day cycle starting from Day 1 of Cycle 2. In the trial, 43% of patients (n=270) had ESR1 mutations detected. The primary endpoint was progression-free survival (PFS) in the ESR1-mutation and intent-to-treat populations as determined by blinded independent central review. Overall survival is the key secondary endpoint.

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

Arvinas Announces FDA Acceptance of the New Drug Application for Vepdegestrant for the Treatment of ESR1m, ER+/HER2- Advanced Breast Cancer

Much-needed new drug approved for deadliest blood cancer (ziftomenib)

2026-04-10T14:40:00.000+05:30

Biomedical research that began at the University of Virginia School of Medicine has yielded a much-needed new treatment for patients with the deadliest form of blood cancer.

The federal Food and Drug Administration has approved the drug ziftomenib for patients with recurring or treatment-resistant acute myeloid leukemia who have a mutation in the NPM1 .. gene. The new medication, taken by mouth once daily, offers a potential treatment for patients who otherwise have no good options.

The drug arose from many years of research by Jolanta Grembecka, Ph.D., and Tomasz Cierpicki, Ph.D., who began the work in 2007 as research assistant professors in UVA's Department of Molecular Physiology and Biological Physics, working closely with John Bushweller, Ph.D., their former postdoctoral mentor. In 2009, both Grembecka and Cierpicki moved to the University of Michigan, where they are professors in the Department of Pathology.

"Ziftomenib is a long-awaited and desperately needed new option for patients for whom other treatments have failed—for patients left with no hope. It's a wonderful achievement by Drs. Grembecka and Cierpicki," said Mark Esser, Ph.D., the head and chief scientific officer of UVA's Paul and Diane Manning Institute of Biotechnology.

"UVA has founded the Manning Institute specifically to advance exactly this type of important research. We are accelerating the development of new treatment and cures for the most complex and difficult diseases to benefit patients everywhere."

About acute myeloid leukemia

Acute myeloid leukemia is a particularly deadly blood cancer primarily seen in people over the age of 68. More than 22,000 Americans develop the condition each year, and more than 11,000 die, according to the American Cancer Society. Overall, the disease accounts for about 1 in 3 cases of blood cancer.

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

M uch-needed new drug approved for deadliest blood cancer

PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA

2026-04-08T14:22:00.000+05:30

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia.

"We are of course disappointed by the FDA's decision to not approve vatiquinone," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for both children and adults living with Friedreich's ataxia. We plan to meet with the FDA to discuss potential steps to address the issues raised in the CRL."

The FDA stated in the CRL that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support NDA resubmission.

About Vatiquinone

Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich's ataxia. Inhibition of 15-LO helps to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately decreasing inflammation and oxidative stress and promoting neuronal survival.1,2,3 Vatiquinone has been evaluated in a number of clinical studies, many focused on pediatric patients, and has demonstrated an impact on mortality risk, and a number of neurological and neuromuscular disease symptoms.

About Friedreich's Ataxia

Friedreich's ataxia (FA) is a rare, physically debilitating, life-shortening, neuromuscular disorder that mainly affects the central nervous system and the heart.4 It is the most common hereditary ataxia (abnormal, uncoordinated movements) and is usually caused by a single genetic defect in the frataxin (FXN) gene that leads to reduced production of frataxin, a mitochondrial protein that is important for cellular metabolism and energy production.4,5 Decreased frataxin levels are associated with mitochondrial iron accumulation and increased oxidative stress, which can lead to cell death through ferroptosis.6,7,8

Symptoms include progressive loss of coordination and muscle strength leading to poor balance and coordination, difficulty speaking, swallowing, and breathing, curvature of the spine, serious heart conditions, diabetes, and hearing and vision impairment.9,10 The severity of symptoms and speed of progression varies between people and some symptoms may not be evident in all. Friedreich's ataxia is usually diagnosed in childhood or adolescence.5,11 Approximately 25,000 people have Friedreich's ataxia globally.

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

PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA

FDA Accepts Shionogi’s Ensitrelvir NDA for Review as the First Oral Therapy for the Prevention of COVID-19 Following Exposure

2026-04-02T09:37:00.005+05:30

Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter “Shionogi”) announced the U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) from Shionogi Inc., a New Jersey-based subsidiary of Shionogi, for ensitrelvir (Generic name: ensitrelvir fumaric acid, Code No.: S-217622), an investigational oral antiviral for the prevention of COVID-19 following exposure to an infected individual. The FDA has set an action date of June 16, 2026 under the Prescription Drug User Fee Act (PDUFA).

The NDA is supported by results from the global, double-blind, randomized, placebo-controlled Phase 3 study, SCORPIO-PEP, which studied ensitrelvir as post-exposure prophylaxis (PEP). If approved, ensitrelvir would be the first and only oral therapy for the prevention of COVID-19 following exposure to an infected individual.1,2

SARS-CoV-2 remains highly transmissible and up to half of people living with an infected individual may develop COVID-19.3,4 The virus is constantly evolving, with novel symptoms reported.5,6 COVID-19 continues to impact daily life and may lead to absences from school and work, may cause long COVID, and in some cases, may progress to severe disease.7-12 Even patients with mild COVID-19 may experience worsening of preexisting chronic conditions.13-17

Following exposure to COVID-19, the best way to avoid its potentially serious and long-term complications is to stop viral replication, which prevents the development of the disease.1,2 There are currently no approved antiviral therapies proven to prevent COVID-19 following exposure.1,2,18 COVID-19 vaccines are received before exposure, and they do not stop viral replication.19,20 Other COVID-19 antivirals are taken following exposure and diagnosis, after viral replication has occurred and disease is already established.21

“Shionogi has a long history of innovation in infectious disease treatment and prevention. Our dedication to this field has led to significant breakthroughs in the development of novel antimicrobials and antivirals for HIV/AIDS, influenza and COVID-19. If approved, ensitrelvir will be the first and only oral therapy to help protect people in the U.S. from COVID-19 following exposure," said Nathan McCutcheon, MBA, President and CEO, Shionogi Inc.

About ensitrelvir

Ensitrelvir is a SARS-CoV-2 main protease inhibitor created through joint research between Hokkaido University and Shionogi. SARS-CoV-2 has an enzyme called the main protease (3-CL), which is essential for the replication of the virus. Ensitrelvir suppresses the replication of SARS-CoV-2 by selectively inhibiting the main protease.

Ensitrelvir, known as Xocova® in countries where it is approved, received emergency regulatory approval in Japan in November 2022 and full approval in March 2024 for the treatment of COVID-19 based on results from SCORPIO-SR, a Phase 3 study conducted in Asia, during the Omicron-dominant phase of the pandemic.

In SCORPIO-SR, ensitrelvir showed both clinical symptomatic efficacy (symptom resolution sustained for at least 24 hours) for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in a predominantly vaccinated population of patients with mild-to-moderate SARS-CoV-2 infection, regardless of risk factors. Most adverse events were mild in severity and no deaths were seen in the study. Among the most common treatment-related adverse events were temporary decreases in high-density lipoprotein and increased blood triglycerides, as observed in previous studies. Results from this study were published in JAMA Network Open.

In 2025, Shionogi submitted two new drug applications in Japan for post-exposure prophylaxis of COVID-19 and for COVID-19 treatment in pediatric patients aged six to under 12 years. The pediatric submission is based on a multicenter, randomized, double-blind, placebo-controlled trial of ensitrelvir in mild-to-moderate COVID-19 patients aged 6 to 12 years in Japan. The study confirmed safety and tolerability and found the pharmacokinetics of ensitrelvir in this age group to be similar to that in adults.

Ensitrelvir became available in Singapore for the treatment of COVID-19 via a Special Access Route application in 2023, and it is currently under regulatory review in Taiwan for the treatment of COVID-19. Ensitrelvir is also under regulatory review with the European Medicines Agency for COVID-19 post-exposure prophylaxis and treatment.22

Ensitrelvir is an investigational drug outside of Japan and Singapore. In addition, the brand name Xocova® has not been approved for use outside of Japan and Singapore and pertains only to the approved drug in Japan and Singapore.

FDA Accepts Shionogi’s Ensitrelvir NDA for Review as the First Oral Therapy for the Prevention of COVID-19 Following Exposure

Achieve Life Sciences Announces FDA Acceptance of Cytisinicline New Drug Application for Treatment of Nicotine Dependence for Smoking Cessation

2026-04-01T15:35:00.008+05:30

Achieve Life Sciences, Inc. (Nasdaq: ACHV), a late-stage specialty pharmaceutical company focused on the global development and commercialization of cytisinicline for treatment of nicotine dependence, today announced that the U.S. Food and Drug Administration (FDA) has accepted the cytisinicline New Drug Application (NDA) for a new treatment for smoking cessation in adults. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) targeted action date of June 20, 2026.

“The FDA’s acceptance of our NDA validates the totality of our product development program,” said Dr. Cindy Jacobs, President and Chief Medical Officer of Achieve Life Sciences. “Our application is supported by a decade of rigorous research and comprehensive data from thousands of participants. We've built compelling scientific and clinical results as a foundation that, if approved, positions cytisinicline to potentially address a significant medical need. We’re eager to engage constructively with the FDA as we progress through the NDA review process.”

The company’s NDA details a comprehensive clinical development program, with more than 2,000 clinical trial participants contributing to the body of evidence. The ORCA-2 and ORCA-3 Phase 3 trials showed cytisinicline administered for either 6 or 12 weeks, alongside standard behavioral support, demonstrated significantly greater smoking abstinence rates by the end of treatment and in long-term abstinence through week 24 as compared to placebo. In addition, safety data will include over 400 participants who have received at least six months of cumulative cytisinicline exposure and over 200 participants receiving at least 1 year of cumulative cytisinicline exposure, with no new safety concerns as reported by the Data Safety Monitoring Committee.

“Smoking is the leading cause of preventable death and disease, claiming the lives of nearly half a million Americans each year, and costing the American economy more than an estimated $600 billion a year,” said Rick Stewart, Chief Executive Officer of Achieve Life Sciences. “The FDA's acceptance of our NDA filing underscores nicotine dependence as an important public health need that demands action. We're energized by this milestone and remain laser-focused on our mission, working toward commercial readiness for the second half of 2026, pending FDA approval. Each year, an estimated 15 million Americans attempt to quit smoking, and we're committed to providing those who are ready to quit a new tool so they can break free from nicotine dependence.”

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

Achieve Life Sciences Announces FDA Acceptance of Cytisinicline New Drug Application for Treatment of Nicotine Dependence for Smoking Cessation

Sentynl Therapeutics Updates, Complete Response Letter (CRL) relating to its New Drug Application (NDA) for On Its NDA for CUTX-101

2026-02-19T07:00:00.001+05:30

Sentynl Therapeutics, Inc. (“Sentynl”), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (“Zydus”), announced today that the U.S. Food and Drug Administration (USFDA) has issued a Complete Response Letter (CRL) relating to its New Drug Application (NDA) for copper histidinate (CUTX-101), intended to treat Menkes disease in pediatric patients.

The USFDA provided findings within the CRL that Sentynl and Zydus will need to address to clarify the path forward. Specifically, the USFDA mentioned a CGMP inspection of the facility where CUTX-101 is manufactured. Zydus recently provided responses to USFDA’s September 2025 re-inspection demonstrating the facility’s CGMP compliance and is awaiting USFDA’s Establishment Inspection Report (EIR). Sentynl will request a meeting with USFDA to discuss the CRL and resubmission of the CUTX-101 NDA. The CRL did not cite any other approvability concerns, nor did it identify any deficiencies in CUTX-101’s efficacy and safety data.

“We recognize the USFDA’s decision and remain dedicated to working with the Agency to clarify next steps. Our commitment to patients is unchanged. We believe in the promise of our therapy and are prepared to address the feedback and pursue resubmission promptly,” said Matt Heck, CEO, Sentynl.

Menkes disease is a rare X-linked recessive pediatric genetic disease that impacts an estimated 1 in 34,810 to as high as 1 in 8,664 live male births. Patients with Menkes disease are born with the inability to absorb dietary copper and subsequently have impaired copper transport across the blood-brain barrier. CUTX-101 is a subcutaneous injectable formulation of copper histidinate that restores copper homeostasis and maintains copper levels in patients with Menkes disease.

The CUTX-101 NDA was initially granted Priority Review by the FDA and is supported by positive topline clinical efficacy results for CUTX-101, demonstrating significant improvement in overall survival for Menkes disease subjects who received early treatment with CUTX-101.

Sentynl Therapeutics Updates On Its NDA for CUTX-101

FDA Approves Linzess (linaclotide) for Children 7 Years and Older with Irritable Bowel Syndrome with Constipation

2026-02-18T07:00:00.004+05:30

The U.S. Food and Drug Administration (FDA) has approved Linzess (linaclotide) capsules for pediatric patients 7 years and older with irritable bowel syndrome with constipation (IBS-C). Linzess is the first treatment approved for IBS-C in pediatric patients.

Disease or Conditions

IBS-C is a common condition affecting children and adolescents characterized by chronic constipation (which occurs when patients have infrequent bowel movements with hard stools that may be difficult or painful to pass), abdominal pain, and bloating. There is no known underlying organic cause and there are typically multiple contributing factors.

Efficacy

The effectiveness of Linzess to treat IBS-C was established in pediatric patients 7 years and older. Linzess for this indication was supported by extrapolation of efficacy from adequate and well-controlled studies in adults and a 12-week double-blind, randomized, parallel-group trial in pediatric patients 7 to 17 years who met modified Rome III criteria for child/adolescent IBS-C. The primary endpoint was the proportion of patients who achieved at least a 30% reduction in abdominal pain and an increase of at least two spontaneous (i.e., naturally occurring) bowel movements per week from baseline for at least 6 weeks of the 12-week treatment period. The efficacy results were consistent with results demonstrated in the adult IBS-C population.

Safety

The safety of Linzess in these clinical studies was similar in adult and pediatric patients. The most common side effect reported in pediatric patients 7 to 17 years with IBS-C was diarrhea. If severe diarrhea occurs, patients should discontinue Linzess and be rehydrated. Patients younger than 2 years can be at risk of serious dehydration and should not take Linzess for any indication, nor should patients with known or suspected mechanical gastrointestinal obstruction (bowel blockage). See the full prescribing information for additional information on risks associated with Linzess.

The recommended dosage for pediatric patients 7 years and older with IBS-C is 145 mcg orally once daily.

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

FDA Approves Linzess (linaclotide) for Children 7 Years and Older with Irritable Bowel Syndrome with Constipation

FDA Files Corcept’s New Drug Application for Relacorilant as a Treatment for Patients with Platinum-Resistant Ovarian Cancer

2026-02-17T07:30:00.000+05:30

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, today announced that the U.S. Food and Drug Administration (FDA) has accepted Corcept’s New Drug Application (NDA) for relacorilant as a treatment for patients with platinum-resistant ovarian cancer. The FDA has assigned a PDUFA date of July 11, 2026 for the application.

Corcept’s NDA is based on positive data from its pivotal Phase 3 ROSELLA and Phase 2 trials. In these trials, patients who received relacorilant plus nab-paclitaxel experienced improved progression-free and overall survival compared to patients who received nab-paclitaxel monotherapy, with no need for biomarker selection. Relacorilant was well-tolerated, consistent with its known safety profile. Importantly, relacorilant conferred its benefit without increasing the safety burden of the patients who received it. The type, frequency and severity of adverse events in the combination arms were similar to those in the nab-paclitaxel monotherapy arms.

“The FDA's acceptance of our NDA brings us closer to offering a much-needed treatment option to patients with this dire disease,” said Joseph Belanoff, M.D., Corcept’s Chief Executive Officer. “Relacorilant has the potential to redefine how platinum-resistant ovarian cancer is treated.”

About Relacorilant

Relacorilant, an oral therapy, is a selective glucocorticoid receptor (GR) antagonist that modulates cortisol activity by binding to the GR but not to the body's other hormone receptors. Corcept is developing relacorilant in ovarian cancer and a variety of other serious disorders, including endogenous hypercortisolism and prostate cancer. Relacorilant is proprietary to Corcept and is protected by composition of matter, method of use and other patents. It has been designated an orphan drug by the FDA and the European Commission (EC) for the treatment of hypercortisolism and by the EC for the treatment of ovarian cancer. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) date of December 30, 2025 for relacorilant as a treatment for patients with hypercortisolism.

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

FDA Files Corcept’s New Drug Application for Relacorilant as a Treatment for Patients with Platinum-Resistant Ovarian Cancer

FDA Approves Caplyta (lumateperone) as an Adjunctive Therapy for the Treatment of Major Depressive Disorder (MDD) in Adults

2026-02-16T07:00:00.003+05:30

In continuation of my update on lumateperone

Johnson & Johnson (NYSE: JNJ) announced the U.S. Food and Drug Administration (FDA) approval of Caplyta® (lumateperone) as an adjunctive therapy with antidepressants for the treatment of major depressive disorder (MDD) in adults. The approval – the first under J&J leadership following its acquisition of Intra-Cellular Therapies, Inc. – provides patients with a safe and effective new treatment option that can enable a path to remission. Caplyta® makes it easy to start and stay on treatment without the need for titration. Weight gain and other metabolic side effects that typically lead to discontinuation of care were similar to placebo. This approval marks the fourth indication for Caplyta®, the first and only FDA-approved treatment for bipolar I and II depression in adults, as an adjunctive and monotherapy; also approved for the treatment of schizophrenia in adults.

MDD, or clinical depression, is one of the most common psychiatric disorders, affecting about 22 million American adults.4,5 While oral antidepressants may offer relief for some, 2 in 3 people living with MDD continue to experience residual symptoms despite treatment, significantly impacting their overall quality of life.6 Beyond its toll on patients’ wellbeing, MDD has a substantial economic burden and is the leading cause of disability in the U.S.7

“Depression is a complex disorder that affects each person differently, underscoring the urgent need for a range of effective and well-tolerated treatment options,” said Roger S. McIntyre, M.D., FRCPC, Professor of Psychiatry and Pharmacology, University of Toronto.a “For people who are still experiencing lingering depressive symptoms while on an antidepressant, adding Caplyta® to a patient’s treatment regimen may offer early improvement, with the potential for remission—the ultimate goal of treatment.”

This approval is based on positive results from two Phase 3, global, double-blind, placebo-controlled trials – Study 501 and 502 – which both met their primary and key secondary endpoints, providing statistically significant and clinically meaningful improvement in depression symptoms compared to an oral antidepressant plus placebo, as measured by Montgomery-Asberg Depression Rating Scale (MADRS) and Clinical Global Impression Scale-Severity index (CGI-S) total scores.1,2

A large separation in total MADRS score was seen between Caplyta® and placebo in Study 501 (-4.9 points, effect size 0.61) and Study 502 (-4.5 points, effect size 0.56), at six weeks. Separation from placebo was seen as early as one week in Study 501 and two weeks in Study 502. Significant reductions in the key secondary endpoint of mean change in total CGI-S scores from baseline were also demonstrated at six weeks in Study 501 (-0.7 points, effect size 0.67) and Study 502 (-0.5 points, effect size 0.51).1,2

In pivotal trials, the Caplyta® safety profile was consistent with the existing body of clinical data in its schizophrenia and bipolar depression I and II indications. No new safety concerns were identified. Weight gain and metabolic changes (lipid and glucose levels), as well as akathisia and restlessness, were similar to placebo. Reports of sexual side effects were not common. The most common side effects of Caplyta® include sleepiness, dizziness, nausea, dry mouth, feeling tired, and diarrhea.

“Major depressive disorder affects millions of Americans, impacting how a person feels, thinks, and acts,” said Michael Pollock, CEO of Depression and Bipolar Support Alliance (DBSA). “DBSA believes that all individuals have the right to direct their own treatment, and we understand that for many people, ongoing antidepressant therapy alone may not offer meaningful relief. The introduction of new treatment options, and continued innovation in mental health, has enabled us to reset expectations for living with depression and offers people hope that achieving lasting wellness and remission is possible.”

Long-term data, evidenced by the 503 open-label extension safety study, showed Caplyta® was safe and well tolerated, consistent with the safety profile of Studies 501 and 502. Patients experienced low risk of weight gain, cardiometabolic effects, and extrapyramidal symptoms.8 Caplyta® also demonstrated the potential to help patients achieve remission. During this 26-week safety study, 80% of patients responded to treatment and 65% of patients experienced remission (defined as MADRS Total score ≤ 10) at 6 months.3

Although its exact mechanism of action is unknown, Caplyta® is characterized by high serotonin 5-HT2A receptor occupancy and moderate amounts of dopamine D2 receptor occupancy at therapeutic doses. Caplyta® does not need dose titration, allowing patients to start treatment at the effective dose of 42 mg.8

“Caplyta® has the potential to become a new standard of care across multiple mental health disorders, including major depressive disorder,” said Bill Martin, Ph.D., Global Therapeutic Area Head, Neuroscience, Johnson & Johnson Innovative Medicine. “This approval is a testament to our nearly 70-year commitment of bringing innovative and differentiated therapies that redefine treatment expectations—and introduce the possibility of remission—to patients living with some of today’s most prevalent and debilitating mental health conditions.”

This additional FDA approval builds upon the established, robust clinical efficacy and proven real-world safety profile of Caplyta® for the treatment of adults living with schizophrenia and for the treatment of depressive episodes associated with bipolar I or II disorder.

A supplemental New Drug Application (sNDA) for Caplyta® with long-term data evaluating the safety and efficacy of the medication for the prevention of relapse in schizophrenia was recently submitted to the FDA. The medication is also being studied for other neuropsychiatric and neurological disorders. Caplyta® is not FDA-approved for these disorders.

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

FDA Approves Caplyta (lumateperone) as an Adjunctive Therapy for the Treatment of Major Depressive Disorder (MDD) in Adults

FDA Approves Zoryve for Atopic Dermatitis in Young Children

2026-02-13T07:00:00.004+05:30

In continuation of my update on Zoryve

The U.S. Food and Drug Administration has approved the supplemental new drug application for Zoryve (roflumilast) cream 0.05 percent for the topical treatment of mild-to-moderate atopic dermatitis in children 2 to 5 years of age.

The once-daily cream can be used anywhere on the body and for any duration. This approval offers an alternative to steroids.

The approval is based on clinical trials that showed Zoryve cream rapidly improved the severity and extent of eczema signs and symptoms, with approximately 40 percent of children achieving a 75 percent improvement from baseline on the Eczema Area and Severity Index. Additionally, more than one-third of participants (35 percent) achieved clinically meaningful improvement in itch intensity in four weeks.

"Young children are particularly vulnerable to the bothersome symptoms of atopic dermatitis, because their immune system and skin barrier are less developed than those of older children and adults," Korey Capozza, founder and executive director at Global Parents of Eczema Research, said in a statement. "This condition doesn't just affect the child's skin -- it can affect the whole family by causing sleep disruption, emotional distress, and social isolation."

Approval of Zoryve was granted to Arcutis Biotherapeutics.

More Information

FDA Approves Zoryve for Atopic Dermatitis in Young Children

Niraparib Plus AAP Improves Survival in mCSPC With HRR Gene Alterations

2026-02-12T07:00:00.003+05:30

In continuation of my update on Niraparib

For patients with metastatic castration-sensitive prostate cancer (mCSPC) with homologous recombination repair (HRR) gene alterations, the addition of niraparib to abiraterone acetate and prednisone (AAP) is beneficial, according to a study published online Oct. 7 in Nature Medicine.

Gerhardt Attard, M.D., Ph.D., from University College London, and colleagues conducted a double-blind trial that evaluated combining niraparib with AAP versus placebo and AAP in mCSPC with HRR gene alterations. A total of 696 patients were randomly assigned to niraparib or placebo (348 each).

Of the patients, 56 percent had BRCA1 or BRCA2 alterations and 78 percent had high-volume metastases. The researchers first observed significant improvement in radiographic progression-free survival in the BRCA subgroup (median not reached at the time of analysis for the niraparib and AAP group versus 26 months for the AAP group; hazard ratio, 0.52). Significant improvement was also seen in the intention-to-treat population (hazard ratio, 0.63). For overall survival, the data were immature but favored niraparib. The incidence of grade 3 or 4 adverse events was 75 and 59 percent in the niraparib and placebo groups, respectively; in the niraparib and AAP group, the most frequent adverse event was anemia (29 percent), with 25 percent of patients needing a blood transfusion.

"For cancers with a mutation in one of the eligible HRR genes, where niraparib has been approved, a doctor should consider a discussion that balances the risks of side effects against the clear benefit to delaying disease growth and worsening symptoms," Attard said in a statement.

Several authors disclosed ties to the biopharmaceutical industry, including Johnson & Johnson, which funded the study.

Abstract/Full Text

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

Niraparib Plus AAP Improves Survival in mCSPC With HRR Gene Alterations - Drugs.com MedNews

Tramadol May Have Slight Effect on Reducing Chronic Pain

2026-02-11T07:00:00.002+05:30

In continuation of my update on Tramadol..

Tramadol may slightly reduce chronic pain but seems to increase the risk for serious and nonserious adverse events, according to a review published online Oct. 7 in BMJ Evidence-Based Medicine.

Jehad Ahmad Barakji, M.D., from the Centre for Clinical Intervention Research in Copenhagen, Denmark, and colleagues conducted a systematic review to examine the benefits and harms of tramadol versus placebo in chronic pain. Data were included from 19 randomized placebo-controlled trials, with 6,506 participants.

The researchers observed evidence of a beneficial effect of tramadol on chronic pain (mean difference numerical rating scale [NRS], −0.93 points) in a meta-analysis and trial sequential analysis, with the effect size below the predefined minimal important difference of 1.0 point on the NRS. There was evidence of a harmful effect of tramadol on serious adverse events (odds ratio, 2.13), which was mainly due to a higher proportion of cardiac events and neoplasms. Due to a lack of data, it was not possible to conduct a meta-analysis on quality of life. The risk for several nonserious adverse events, including nausea, dizziness, constipation, and somnolence, was increased with tramadol (number needed to harm: seven, eight, nine, and 13, respectively).

"In the United States, the number of opioid-related overdose deaths increased from 49,860 in 2019 to 81,806 in 2022," the authors write. "Given these trends and the present findings, the use of tramadol and other opioids should be minimized to the greatest extent possible."

Abstract/Full Text

Tramadol May Have Slight Effect on Reducing Chronic Pain - Drugs.com MedNews

High Flavanol Consumption May Counteract Sitting-Induced Endothelial Dysfunction

2026-02-10T07:00:00.002+05:30

In continuation of my update on Flavanol

Consuming high-flavanol foods and drinks may reduce some of the impact of prolonged sitting on the vascular system, according to a study published online Oct. 29 in the Journal of Physiology.

Alessio Daniele, from the University of Birmingham in the United Kingdom, and colleagues investigated whether intake of dietary flavanols prior to a two-hour sitting bout can preserve upper- and lower-limb endothelial function in high- and low-fit individuals. The analysis included 40 young, healthy men (20 high-fit; 20 low-fit) who completed a two-hour sitting trial after consuming either a high-flavanol (150 mg epicatechin) or low-flavanol (<6 mg epicatechin) cocoa intervention.

The researchers found that sitting significantly reduced flow-mediated dilation (FMD) in the superficial femoral artery and brachial artery. Sitting also increased diastolic blood pressure (BP) in both fitness groups. High-flavanol consumption prevented FMD declines in both arteries, with no effects on BP. There were significant decreases in shear rate and blood flow in both arteries in both fitness groups with sitting, with no effects of the flavanol intervention. Sitting was also associated with declines in tissue oxygenation (TOI), detectable within 10 minutes, and impaired TOI desaturation and speed of reperfusion during hyperemia two hours after sitting, with no effects of flavanols.

"Given how common sedentary lifestyles have become and the increased risk this can have to vascular health, using flavanol-rich food and drink, especially in combination with breaking up periods of inactivity by going for a short walk or standing up, could be a good way to enhance long-term health, no matter the individual's fitness level," coauthor Catarina Rendeiro, also from University of Birmingham, said in a statement.

Abstract/Full Text

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

High Flavanol Consumption May Counteract Sitting-Induced Endothelial Dysfunction - Drugs.com MedNews

Intranasal Insulin, Empagliflozin Have Promising Effects on Cognition

2026-02-09T07:00:00.003+05:30

In continuation of my update Empagliflozan

Intranasal insulin (INI) and empagliflozin are safe and have promising effects on cognition, according to a study published online Oct. 7 in Alzheimer's & Dementia.

Jennifer M. Erichsen, from the Wake Forest University School of Medicine in Winston-Salem, North Carolina, and colleagues conducted a phase 2, 2x2 factorial double-blinded randomized trial involving 47 participants with mild cognitive impairment or early Alzheimer disease or who were amyloid-positive. Participants received INI, the sodium-glucose cotransporter-2 inhibitor empagliflozin, both, or placebo for four weeks. Treatment-related adverse events were examined as the primary outcome.

The researchers found that for all groups, treatment-related adverse events were mild and similar. They also found an increase in modified Preclinical Alzheimer's Cognitive Composite-5, modulated fractional anisotropy and cerebral blood flow, and reduced plasma glial fibrillary acidic protein with INI. Lower cerebrospinal fluid tau and modulated cerebral blood flow were seen with empagliflozin. Immune/inflammatory/neurovascular markers were moderated by both agents.

"For the first time, we found that empagliflozin, an established diabetes and heart medication, reduced markers of brain injury while restoring blood flow in critical brain regions. We also confirmed that delivering insulin directly to the brain with a newly validated device enhances cognition, neurovascular health and immune function. Together, these findings highlight metabolism as a powerful new frontier in Alzheimer's treatment," coauthor Suzanne Craft, Ph.D., also from the Wake Forest University School of Medicine, said in a statement.

Aptar Pharma provided the intranasal delivery devices.

Abstract/Full Text (subscription or payment may be required)

Intranasal Insulin, Empagliflozin Have Promising Effects on Cognition

AHA: Low-Dose Aspirin Use Linked to Lower ASCVD Outcomes in Type 2 Diabetes

2026-02-06T07:00:00.002+05:30

In continuation of my update on aspirin

For adults with type 2 diabetes, low-dose aspirin (ASA) use is associated with a lower risk for myocardial infarction (MI) and stroke, with greater benefit seen with high-frequency use, according to a study presented at the American Heart Association Scientific Sessions 2025, held from Nov. 7 to 10 in New Orleans.

Aleesha Kainat, M.D., from the University of Pittsburgh Medical Center, and colleagues examined the impact of ASA use and adherence frequency on atherosclerotic cardiovascular disease (ASCVD) outcomes among adult patients with diabetes with a moderate or high 10-year ASCVD risk score. Baseline characteristics were balanced between ASA users and nonusers in a propensity score-matched analysis.

Among 11,618 patients, 88.6 and 53.15 percent were ASA and statin users, respectively, at any point during 10-year follow-up. The researchers found that the cumulative incidence of MI, stroke, and 10-year all-cause mortality was significantly lower in the ASA group versus the no-ASA group (42.4 versus 61.2 percent, 14.5 versus 24.8 percent, and 33 versus 50.7 percent, respectively). Compared with no ASA use, any ASA use was associated with significantly lower hazards of MI and ischemic stroke, with greater benefit seen in the high-frequency use group (hazard ratios, 0.54 and 0.47, respectively). Consistent benefit was seen for ASA across glycemic strata, although the magnitude of benefit decreased with worse glycemic control. In better controlled groups, the reduction in mortality was also more pronounced.

"We were somewhat surprised by the magnitude of the findings," Kainat said in a statement. "People with type 2 diabetes and a higher risk of CVD who reported taking low-dose aspirin were much less likely to have had a heart attack, stroke, or death over 10 years when compared to similar individuals who did not report taking low-dose aspirin. That benefit was greatest for those who took aspirin consistently, throughout most of the follow-up time

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

AHA: Low-Dose Aspirin Use Linked to Lower ASCVD Outcomes in Type 2 Diabetes - Drugs.com MedNews

Pazopanib Well-Tolerated in Young With Primary Multimetastatic Ewing Sarcoma

2026-02-05T07:00:00.001+05:30

In continuation of my update on Pazopanib

For young patients with primary multimetastatic Ewing sarcoma, pazopanib is well-tolerated and effective, according to a study published online Oct. 22 in Frontiers in Oncology.

Noting that pazopanib has been proposed as an effective salvage regimen for soft tissue sarcoma, including extraosseous Ewing sarcoma, Anna Raciborska, M.D., Ph.D., from the Institute of Mother and Child in Warsaw, Poland, and colleagues examined this approach for young patients with primary multimetastatic bone Ewing sarcoma. Eleven patients received standard first-line treatment in parallel with pazopanib.

The researchers found that pazopanib was administered for an average of 1.7 years, throughout the treatment period and after its completion (paused during the surgical procedure). Patients' median age was 14.2 years at the beginning of pazopanib treatment. Five patients underwent surgery on the primary tumor; 10 received concurrent radiation therapy and three underwent autologous hematopoietic stem cell transplantation. No significant toxicities were observed. One, two, and one patients progressed, had relapse, and died (9.1, 18.2, and 9.1 percent, respectively). With a median follow-up of 2.6 years, 10 patients (90.9 percent) were alive. For the whole group, estimated two-year event-free and overall survival were 68.2 and 85.7 percent, respectively.

"Survival rates were higher than in historical controls, suggesting it may extend lives and, importantly, do so without adding severe toxicity," Raciborska said in a statement. "Moreover, the quality of life of treated children was good."

More : Abstract/Full Text

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

Pazopanib Well-Tolerated in Young With Primary Multimetastatic Ewing Sarcoma - Drugs.com MedNews

Existing drug, Ulipristal acetate- could reduce breast cancer risk in pre-menopausal women

2026-02-04T07:30:00.003+05:30

In continuation of my update on Ulipristat

NYU Langone Health researchers found that a type of cell death caused by a buildup of highly reactive molecules suppresses lung tumor growth.

The process, called ferroptosis, evolved to let the body signal for self-destruction of cells that are overly stressed for various reasons. This includes cancer cells, but they in turn evolved to have mechanisms that counter ferroptosis so they can continue their uncontrolled growth despite the stress it creates.

Published in Nature, the new study showed that an experimental treatment blocked the action in cancer cells of a protein called ferroptosis suppressor protein 1 (FSP1) to reduce by up to 80% tumor growth in mice with lung adenocarcinoma (LUAD).

Lung cancer is the leading cause of cancer death worldwide, with LUAD the most common lung cancer among nonsmokers, making up about 40% of cases.

"This first test of a drug that blocks ferroptosis suppression highlights the importance of the process to cancer cell survival and paves the way for a new treatment strategy," said senior study author Thales Papagiannakopoulos, Ph.D., an associate professor in the Department of Pathology at the NYU Grossman School of Medicine.

Harmful reactions

Ferroptosis kills cells by building up levels of iron, which generates highly reactive molecules formed from oxygen, water, and hydrogen peroxide called reactive oxygen species (ROS).

Important for normal cell signaling, ROS also often cause oxidative stress, a disease mechanism in which ROS oxidize (add oxygen molecules to) delicate proteins and DNA to pull them apart. ROS damage fats making up the outer layers of cells to cause cell death and tissue damage.

For the new study, the research team genetically engineered mice to delete the FSP1 gene in lung cancer cells, which led to increased cancer cell death and significantly smaller tumors.

The team also found that a potent, relatively new drug type, an FSP1 inhibitor called icFSP1, improved overall survival of lung tumor-bearing mice, nearly to the extent seen in mice with lung tumors genetically engineered to lack the FSP1 gene.

Their work also showed that Fsp1 is likely a better target for future drugs than another ferroptosis suppressor, glutathione peroxidase 4 (GPX4), which has been studied longer.

FSP1 was shown in the new study to play a greater role in blocking ferroptosis in lung cancer cells specifically, and a smaller role than GPX4 in normal cell functions (likely fewer side effects). Unlike with GPX4, increased FSP1 levels were also found to track with poorer survival in human LUAD patients.

"Our future research will focus on optimizing FSP1 inhibitors and investigating the potential of harnessing ferroptosis as a treatment strategy for other solid tumors, such as pancreatic cancer," said lead study author Katherine Wu, an MD/Ph.D. student working in the Papagiannakopoulos lab.

"We aim to translate these findings from the lab into novel clinical therapies for cancer patients."

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

Existing drug could reduce breast cancer risk in pre-menopausal women

FDA Issues Complete Response Letter for Biohaven's Vyglxia (troriluzole) New Drug Application for Spinocerebellar Ataxia

2026-02-03T07:00:00.005+05:30

Biohaven Ltd. (NYSE: BHVN) ("Biohaven"), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, announced it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) seeking approval of Vyglxia (troriluzole) for the treatment of spinocerebellar ataxia (SCA).

Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven said, "We are extremely disappointed on behalf of patients by this action from the Office of Neuroscience at FDA. Beyond substantial evidence of safety and efficacy, patients with rare diseases also deserve an efficient, fair and flexible regulatory process that aligns with the urgency of their high unmet medical needs. Such an approach has been mandated by Congress to empower the FDA with maximum regulatory flexibility for rare disease. As a company, we are committed to advancing innovative treatments and remain dedicated to SCA patients despite all the challenges associated with pursuing therapies for rare diseases. Real-world evidence is an important research approach to assessing and delivering new therapies for complex rare diseases but, despite FDA policy initiatives supporting such tools, the front-line review divisions are not yet embracing FDA policy for the use of real-world evidence or the application of regulatory flexibility for rare disease."

Jeremy Schmahmann, M.D., Professor of Neurology at Harvard Medical School and Founding Director of the Ataxia Unit at Massachusetts General Hospital (MGH), added, "Patients with SCA and clinicians who treat them deserve to be heard on this important NDA filing. There is too much at stake for patients. The FDA decision not to listen to disease experts and respect the patient perspective before taking action represents a misstep in the due process, and a failure to deploy regulatory flexibility to evaluate benefit:risk of a medication that has proven to be safe and effective for this rare, debilitating neurodegenerative disease that has no current treatment."

Dr. Coric added, "The development of Vyglxia® (troriluzole) by Biohaven embodies a strong scientific process and deep commitment that is critical to bringing safe and effective treatments to patients with rare diseases like SCA. Our efforts over eight years, included developing the f-SARA scale in collaboration with the FDA and a real-world evidence study in SCA that showed Vyglxia achieved highly consistent, sustained, robust and clinically meaningful treatment effects with a safe, once-daily oral pill that slowed disease progression by 50-70%. The NDA also included data showing Vyglxia reduced the risk of falls and delayed time to becoming wheelchair bound. The leading SCA experts in the United States directly communicated their support of the troriluzole data to the FDA but unfortunately the Office of Neuroscience's inability to collaboratively engage with Biohaven, the patient community and leading experts leave us with concerns about the lack of regulatory flexibility that is being applied for rare, life-threatening conditions. There are a number of common sense solutions and regulatory tools that the Office of Neuroscience could have applied including a fair hearing of the drug's efficacy and safety risks at an Advisory Committee of experts and patients, post-marketing studies, labelling limitations or an accelerated approval pathway. Patients are waiting and the certainty of disease progression for SCA patients far outweighs any residual uncertainty regarding potential design bias or interpretation of study data, especially when the primary outcome measure was achieved in a study protocol and statistical analysis plan that was reviewed by the FDA prior to data analysis. SCA patients deserved approval of Vyglxia and certainly a more balanced interpretation of benefit:risks."

Biohaven remains committed to working with the FDA to find a path forward for its NDA for Vyglxia and plans to meet with the FDA to discuss potential next steps.

Prioritizing Clinical-Stage, Innovative Assets

Biohaven will prioritize resources to focus all its R&D resources on other key programs from its diversified portfolio. Consistent with Biohaven's enduring commitment as a patient-first drug developer, the company's pipeline is focused on a range of disease indications which have limited or no treatment options and are long overdue for therapeutic innovation.

Bruce Car Ph.D., Chief Scientific Officer at Biohaven, commented, "As drug developers we expect setbacks and our diversified portfolio affords us the opportunity to pivot to other key programs. We remain as resilient as ever in following science in order to make a difference in the lives of people with debilitating diseases. Much important work remains, and we are energized and focused on achieving the critical milestones that lie ahead, mindful that days matter and patients are waiting."

Biohaven is initiating strategic portfolio and cost optimization across multiple programs and will focus forward-looking spend on restructuring of business priorities to achieve an approximately 60% reduction in annual direct R&D spend (which excludes personnel and SBC). This may include pausing or delaying non-priority programs to maintain its cash runway to focus on the priority clinical-stage programs over the next year.

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

https://www.drugs.com/nda/vyglxia_251105.html

Combination treatment may help cut lifelong ibrutinib for chronic lymphocytic leukemia patients

2026-02-02T07:00:00.005+05:30

In continuation of my update on ibrutinib

Chronic lymphocytic leukemia (CLL) is the most prevalent adult leukemia in the Western Hemisphere, affecting approximately 200,000 people in the United States.

A study appearing in Clinical Cancer Research found that adding the investigational antibody ianalumab (VAY736) to ibrutinib (Imbruvica) allowed some patients with CLL to discontinue daily therapy and potentially improve their quality of life.

Ianalumab targets the B-cell activating factor receptor (BAFR) and ibrutinib belongs to a class of therapeutics called Bruton's tyrosine kinase inhibitors (BTKi).

"BTKis have revolutionized CLL treatment, but patients typically stay on them indefinitely and the therapy can cause long-term toxicity," said John C. Byrd, MD, senior author of the study, who was chair of the Department of Internal Medicine at the University of Cincinnati College of Medicine.

Taking ibrutinib serves as a daily reminder of illness, which many patients find psychologically burdensome, added Byrd, who is currently director of the UPMC Hillman Cancer Center and associate vice chancellor for cancer affairs at the University of Pittsburgh School of Medicine.

Byrd, Kerry A. Rogers, MD, associate professor from The Ohio State University, and other investigators tested this treatment approach that could potentially help patients with CLL avoid long-term therapy.

They selected ianalumab because preclinical studies from Byrd's lab demonstrated superior activity in combination with BTKi drugs when tested against CLL.

Ianalumab blocks signals from the BAFR, preventing cancerous B cells from surviving and maturing, and also marks the cells so the natural killer (NK) cells in the immune system can destroy them.

"We tested whether this antibody could eliminate residual disease and even resistant clones, offering patients a chance to come off therapy," Byrd explained.

Byrd and colleagues conducted a Phase I, open-label, multicenter trial enrolling 39 patients who did not have complete remission on ibrutinib or had developed resistance mutations. Participants received intravenous ianalumab every two weeks alongside a standard dose of ibrutinib for up to eight cycles.

The study evaluated safety, tolerability, and antitumor activity, as well as whether the combination could deepen responses enough to discontinue BTKi therapy.

The combination therapy had no dose-limiting toxicities, according to Byrd. Grade 3 or greater adverse events occurred in 41% of patients, primarily low levels of neutrophils. Overall response was nearly 60%, and 43.6% had undetectable measurable residual disease (uMRD) in blood or bone marrow.

Byrd noted that 17 patients were able to stop ibrutinib and remain off therapy for 12 to 24 months. Biomarker analyses indicated that ianalumab enhanced NK and T-cell activation, supporting its proposed mechanism of action.

Thirteen patients had uMRD in both blood and bone, while four patients had uMRD solely in bone, which Byrd noted as deep responses. "Patients who experience deep responses can stop daily medication, a powerful shift that removes the constant reminder of cancer," said Byrd.

"Taking a medicine every day can be a reminder of sickness for patients, so it is very symbolic for patients with blood cancers to be able to go off therapy," Byrd noted.

The findings have important implications for patients living with CLL, Byrd added. Data showed that this approach could help patients avoid the cumulative toxicity associated with lifelong BTKi therapy.

The infection rates in the patients in this trial were lower than those historically reported with single-agent BTKi therapy, suggesting that adding ianalumab did not increase infection risk.

"These results point to potentially using fixed-duration combination therapy to achieve remission and reduce the burden of continuous treatment," Byrd said.

The limitation of this study is the small sample size and lack of long-term follow-up. "A larger trial is needed to confirm whether this approach can become a standard strategy for reducing BTKi treatment duration," said Byrd.

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

Combination treatment may help cut lifelong ibrutinib for chronic lymphocytic leukemia patients

FDA Approves Kygevvi (doxecitine and doxribtimine) for the Treatment of Thymidine Kinase 2 Deficiency

2026-01-31T07:00:00.004+05:30

UCB (Euronext Brussels: UCB), a global biopharmaceutical company, today announced that Kygevvi has been granted approval by the U.S. Food and Drug Administration (FDA) for the treatment of adults and pediatric patients living with thymidine kinase 2 deficiency (TK2d), with an age of symptom onset on or before 12 years.1 It is the first and only approved treatment for these patients living with TK2d.

TK2d is an ultra

Doxecitine/doxribtimine

-rare, life-threatening, genetic mitochondrial disease characterized by progressive (worsening over time) and severe muscle weakness (myopathy) with no approved treatment options beyond supportive care until now.2,3,4,5 It is often fatal, with those experiencing initial symptoms on or before the age of 12 years facing a high risk of premature death (often occurring within 3 years after symptom onset).6 It is estimated that the worldwide prevalence of TK2d is 1.64 [0.5, 3.1] cases per 1,000,000 people.7

“The approval of doxecitine and doxribtimine represents a pivotal moment for the TK2d community who previously had no FDA-approved treatment options for this rare genetic mitochondrial disease beyond supportive [palliative] care,” said Donatello Crocetta, Chief Medical Officer at UCB. “We extend heartfelt thanks to the patients, families and friends, advocates, healthcare providers and dedicated clinical trial teams who have partnered with us on this important journey.”

“It’s hard to overstate the importance of this FDA approval for those diagnosed with TK2d. This is an ultra-rare disease community in dire need of treatment options. For too long, caregivers and their families have had to endure the burden of this disease," said Kristen Clifford, United Mitochondrial Disease Foundation President and CEO. “Having the first-ever FDA-approved therapy for TK2d in this patient population not only meets a critical medical need - it represents something greater - hope for the future."

“I’ve been studying mitochondrial diseases for more than three decades and have witnessed firsthand the impact TK2d has on patients and their families. We have been waiting for an approved treatment for many years, and this approval marks a significant milestone in how we can support and manage this debilitating condition,” said Dr. Michio Hirano, Professor of Neurology and Chief of the Division of Neuromuscular Medicine at Columbia University Irving Medical Center.

Supporting data

The Kygevvi approval is supported by safety and efficacy data from one Phase 2 clinical study, two retrospective chart review studies, and an expanded access use program*.1,8,9,10,11 These studies included a total of 82 unique patients treated with Kygevvi or pyrimidine nucleosides with an age of TK2d symptom onset ≤12 years. Efficacy was assessed by comparing overall survival in these pediatric and adult treated patients to an external control group of untreated patients who were matched to treated patients using age of TK2d symptom onset (≤ 2 years or >2 to ≤ 12 years).1 A total of 78 matched pairs were identified. The results showed that survival time from treatment start was improved; treatment reduced the overall risk of death from treatment start by approximately 86% (95% CI: 61%, 96%).1 Of the 78 treated patients included in the survival analysis, the median age of TK2d symptom onset was 1.5 years (range: 0.01 to 12 years).1 The median duration of treatment was 4 years (range: 1 day to 12 years) and the median dose received was 762 mg/kg/day (range: 260 to 800 mg/kg/day).1

The most common adverse reactions (incidence ≥5%) are diarrhea, abdominal pain (including abdominal pain upper), vomiting, alanine aminotransferase increased (ALT), and aspartate aminotransferase increased (AST).1

A regulatory review of doxecitine and doxribtimine is currently underway by the EMA (European Medicines Agency), and further regulatory submissions are planned. Kygevvi is currently not approved for use in any indication by any regulatory authority outside of the U.S. UCB expects Kygevvi to be commercially available in the U.S. in Q1, 2026. To further its mission of equitable care, UCB will provide a personalized support program for Kygevvi that places the needs of patients and caregivers at the forefront.

In the U.S., Kygevvi received Orphan Drug, Breakthrough, Priority Review and Rare Pediatric Disease designations from the FDA.12,13 With this approval by FDA, UCB was awarded a Rare Pediatric Disease Priority Review Voucher (RPDPRV) redeemable for a priority review for a future marketing application.

About Kygevvi

Kygevvi is a combination of doxecitine and doxribtimine, both pyrimidine nucleosides, indicated for the treatment of thymidine kinase 2 deficiency (TK2d) in adults and pediatric patients with an age of symptom onset on or before 12 years.1 Administration of Kygevvi is intended to incorporate the pyrimidine nucleosides, deoxycytidine and deoxythymidine, into skeletal muscle mitochondrial DNA.1 This action restores mitochondrial DNA copy number in TK2d mutant mice.1

Important safety information for Kygevvi1

Increase in Liver Transaminases

Elevated liver transaminase [alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST)] levels were reported in patients treated with Kygevvi. Obtain baseline liver transaminase (ALT, AST) and total bilirubin levels in patients prior to treatment initiation with Kygevvi. If signs or symptoms consistent with liver injury are observed, interrupt treatment with Kygevvi until liver transaminase (ALT, AST) and total bilirubin levels have either returned to baseline or stabilized at a new baseline value. Consider permanently discontinuing Kygevvi if signs or symptoms consistent with liver injury persist or worsen. Monitor liver transaminases and total bilirubin levels yearly and as clinically indicated.

Gastrointestinal Adverse Reactions

Diarrhea and vomiting leading to hospitalization, dose reduction, and permanent discontinuation were reported in patients treated with Kygevvi. Based on the severity of the diarrhea and/or vomiting, reduce the dosage of Kygevvi or interrupt treatment until diarrhea and/or vomiting improves or returns to baseline. Consider restarting Kygevvi at the last tolerated dose, and increase the dose as tolerated. For persistent or recurring diarrhea and/or vomiting, consider discontinuing Kygevvi permanently and provide supportive care with electrolyte repletion as clinically indicated.

https://en.wikipedia.org/wiki/Doxecitine/doxribtimine

FDA Approves Kygevvi (doxecitine and doxribtimine) for the Treatment of Thymidine Kinase 2 Deficiency

FDA Approves Epioxa (riboflavin 5’-phosphate) Ophthalmic Solution for the Treatment of Keratoconus

2026-01-30T07:00:00.003+05:30

Glaukos Corporation, an ophthalmic pharmaceutical and medical technology company focused on novel therapies for the treatment of glaucoma, corneal disorders, and retinal diseases, announced the U.S. Food and Drug Administration (FDA) approval of its Epioxa™ HD / Epioxa™ (“Epioxa”) New Drug Application (NDA). Epioxa is a groundbreaking advancement in corneal cross-linking for the treatment of keratoconus, a rare, sight-threatening disease that is currently far too often undiagnosed and untreated.

Epioxa represents a transformative innovation in keratoconus care, offering an incision-free alternative to traditional corneal cross-linking procedures as it does not require the removal of the corneal epithelium, the outermost layer of the front of the eye. This novel, oxygen-enriched topical therapeutic, bioactivated by UV light, is designed to eliminate the pain associated with removal of the epithelium, streamline the procedure, and minimize recovery, all while delivering clinically meaningful outcomes and exceptional value to patients, providers, and the healthcare system. Epioxa is based on two formulations, Epioxa HD and Epioxa, that are sequentially administered during the cross-linking procedure followed by UV activation in an oxygen-enriched environment.

“The FDA approval of Epioxa ushers in a new standard-of-care for patients suffering from keratoconus with the first FDA-approved topical drug therapy that does not require removal of the corneal epithelium,” said Thomas Burns, Glaukos chairman and chief executive officer. “Epioxa is designed to significantly improve patient comfort and minimize recovery time, representing a game-changing new treatment for patients suffering from keratoconus. We appreciate the clinical investigators and study participants in the clinical trials for their instrumental roles in helping us reach this important advancement. This approval marks a major milestone in our mission to improve patient access to sight-saving therapies, and we are excited to bring this transformative therapy to market for the benefit of patients.”

“Keratoconus is currently an underdiagnosed and undertreated disease. For keratoconus patients who are fortunate enough to be diagnosed, the current standard-of-care requires removal of the epithelium, the top layer of the cornea. The pain and extended healing time associated with the current surgical procedure are major barriers to adoption,” said W. Barry Lee, MD, corneal specialist at Eye Consultants of Atlanta and President of the Cornea Society. “As an incision-free treatment that does not require removal of the epithelium, I expect the newly approved Epioxa treatment to address both of these major concerns.”

The FDA approval of Epioxa is based on results from two prospective, randomized, multicenter, double-masked, Phase 3 pivotal trials that randomized a total of over 400 patients. Both trials successfully achieved their pre-specified primary efficacy endpoints and demonstrated favorable tolerability and safety profiles.

Glaukos intends for Epioxa to be commercially available in the first quarter of 2026. With this approval, Glaukos plans to make substantial investments in patient awareness and access while addressing the longstanding challenges of underdiagnosis and undertreatment that have affected this rare disease patient community. These efforts will be focused on supporting patients and families across every step of the journey, from awareness to diagnosis and treatment. Key initiatives include streamlined patient access support programs, a co-pay assistance program to reduce financial barriers, integrated healthcare professional (HCP) and patient-centric strategies to improve education and engagement, and broad awareness and detection programs aimed at earlier and more widespread screening for and diagnosis of the disease.

Keratoconus is a debilitating eye condition characterized by progressive thinning and weakening of the cornea that is often most aggressively advancing in patients under the age of 30. If left untreated, keratoconus can lead to loss of visual function and even blindness and is one of the leading causes of corneal transplants (penetrating keratoplasty) in the United States. Approximately 90% of cases of keratoconus are bilateral and as many as 20% of untreated keratoconus patients ultimately require a corneal transplant. Conventional keratoconus treatments such as eyeglasses or contact lenses address visual symptoms only and do not slow or halt underlying disease progression.

Glaukos’ first-generation corneal cross-linking therapy, known as Photrexa® Viscous / Photrexa®, which requires removal of the corneal epithelium, received U.S. FDA approval in 2016 as an orphan drug and has since been the first-and-only FDA-approved corneal cross-linking therapy for the treatment of keratoconus. There are more than 300 peer-reviewed publications supporting the performance and safety of Glaukos’ cross-linking therapies.

For more information about Epioxa HD / Epioxa and Full Prescribing Information, please visit www.Epioxa.com.

FDA Approves Epioxa (riboflavin 5’-phosphate) Ophthalmic Solution for the Treatment of Keratoconus