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FDA Approves First Generic Mifepristone as Takeda Discontinues Cell Therapy Research Programs

Drug Approval
  • The FDA has quietly approved the first generic version of mifepristone tablets, marking a significant regulatory milestone that could broaden access and lower costs for the medication commonly used in combination with misoprostol for medication abortions.
  • Takeda Pharmaceuticals announced it will discontinue all cell therapy research programs as part of a strategic shift to focus resources on areas with stronger potential for scientific and commercial impact, including oncology, rare diseases, and plasma-derived therapies.
  • SciSafe has expanded its biopharma storage infrastructure with a new facility in East Brunswick, New Jersey, designed to support clinical and commercial-scale storage of advanced therapies under strict temperature-controlled conditions.

Health Canada Approves AGAMREE as First Treatment for Duchenne Muscular Dystrophy in Canada

Drug ApprovalRare Disease
  • Health Canada has approved AGAMREE (vamorolone) as the first treatment for Duchenne muscular dystrophy in patients aged 4 years and older, marking a historic milestone for Canadian DMD care.
  • The approval was granted under Priority Review and is based on the pivotal VISION-DMD study, where AGAMREE met its primary endpoint with significant improvement in Time to Stand velocity versus placebo (p=0.002).
  • Unlike traditional corticosteroids, AGAMREE demonstrates comparable efficacy while showing no restriction of growth and no negative effects on bone metabolism, potentially offering a better-tolerated treatment option.
  • The Canadian Neuromuscular Disease registry estimates more than 800 boys and young men are living with DMD in Canada, representing a significant unmet medical need now addressed by this approval.

FDA Approves Genentech's Tecentriq Plus Lurbinectedin as First-Line Maintenance Therapy for Extensive-Stage Small Cell Lung Cancer

Drug Approval
  • The FDA has approved Genentech's Tecentriq in combination with lurbinectedin as the first and only combination therapy for first-line maintenance treatment of extensive-stage small cell lung cancer.
  • The Phase III IMforte study demonstrated that the combination reduced the risk of disease progression or death by 46% and the risk of death by 27% compared to Tecentriq alone.
  • The approval addresses a critical unmet need in ES-SCLC, a highly aggressive disease with limited treatment options and high relapse rates.
  • The regimen is now recommended as a category 2A and preferred option in National Comprehensive Cancer Network Guidelines for SCLC maintenance treatment.

A Phase III, Open-Label Study of Maintenance Lurbinectedin in Combination With Atezolizumab Compared With Atezolizumab in Participants With Extensive-Stage Small-Cell Lung Cancer

NCT05091567Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 11/18/2021

FDA Approves Second Generic Mifepristone Despite Trump Administration Safety Review

Drug Approval
  • The FDA approved a new generic version of mifepristone manufactured by Evita Solutions LLC, marking the second generic approval for the abortion medication.
  • The approval occurred while HHS Secretary Robert F. Kennedy Jr. has been conducting a safety review of mifepristone and promising Republican officials a comprehensive evaluation.
  • Anti-abortion leaders and Republican Senator Josh Hawley criticized the decision, calling it a failure of FDA leadership and undermining of state abortion restrictions.
  • The generic approval follows standard regulatory procedures, with the FDA stating it has "very limited discretion" in approving generic drugs that demonstrate identical formulation to brand-name versions.

Hologic Receives FDA Clearance for Automated GI Bacterial Pathogen Detection Assays

Drug Approval
  • Hologic received FDA 510(k) clearance and European CE marking for its Panther Fusion Gastrointestinal Bacterial and Expanded Bacterial Assays, marking the company's entry into GI pathogen diagnostics.
  • The molecular tests rapidly detect common bacterial pathogens causing infectious gastroenteritis, including Salmonella, Campylobacter, Shigella, E. coli, Vibrio, Yersinia and Plesiomonas, addressing a significant healthcare burden with nearly 200 million annual US cases.
  • The customizable mini-panel format allows laboratories to run tests individually or in combination, reducing labor-intensive traditional culture methods while supporting faster diagnosis and antimicrobial stewardship.
  • The assays integrate with Hologic's established Panther Fusion System platform, expanding the company's molecular diagnostics menu beyond women's health and sexually transmitted infections into infectious gastroenteritis testing.

Wockhardt Submits First Indian-Developed Antibiotic to US FDA for Multidrug-Resistant UTI Treatment

Drug Approval
  • Wockhardt has submitted a New Drug Application to the US FDA for Zidebactam-Cefepime injection, marking the first NDA submission for a drug fully discovered and developed by an Indian pharmaceutical company.
  • The novel antibacterial agent targets complicated urinary tract infections caused by multidrug-resistant Gram-negative bacteria, addressing a significant global health burden with over 8 million cases reported annually in the US and EU.
  • The submission follows successful completion of a pivotal Phase III global clinical trial conducted across 64 sites in nine countries, with the drug demonstrating efficacy against carbapenem-resistant infections.
  • Zidebactam-Cefepime represents a first-in-class β-lactam enhancer combination with a novel mechanism of action to combat difficult-to-treat infections that remain unresponsive to currently approved antibiotics.

FDA Grants Priority Review for Leniolisib in Children with Rare Immunodeficiency APDS

Drug ApprovalRare Disease
  • The FDA has accepted Pharming Group's supplemental New Drug Application for leniolisib in children aged 4-11 years with APDS, granting Priority Review with a target decision date of January 31, 2026.
  • If approved, leniolisib would become the first and only treatment specifically indicated for children with activated phosphoinositide 3-kinase delta syndrome, a rare primary immunodeficiency affecting 1-2 people per million worldwide.
  • The application is based on positive Phase III data showing improvements in lymphadenopathy and naïve B cell counts over 12 weeks, indicating correction of the underlying immune defect.
  • APDS is a progressive disease that typically begins in early childhood, causing immune dysregulation, recurrent infections, and potentially permanent lung damage and lymphoma.

FDA Approves Remibrutinib as First Oral BTK Inhibitor for Chronic Spontaneous Urticaria

Drug Approval
  • The FDA has approved remibrutinib (Rhapsido) as the first oral Bruton's tyrosine kinase inhibitor for treating chronic spontaneous urticaria in adults who remain symptomatic despite antihistamine therapy.
  • Phase 3 REMIX trials demonstrated significant improvements in itch, hives, and urticaria activity scores, with well-controlled disease achieved as early as week 2 and approximately one-third of patients experiencing complete symptom absence by week 12.
  • The oral medication offers a convenient alternative to injectable treatments for the estimated 1.7 million Americans with CSU, more than half of whom remain symptomatic despite high-dose antihistamines.
  • Remibrutinib targets BTK protein activation to inhibit histamine release from mast cells and basophils, representing a novel therapeutic approach for this immune-mediated condition.

A Phase 3 Study of Efficacy and Safety of Remibrutinib in the Treatment of CSU in Adults Inadequately Controlled by H1 Antihistamines

NCT05030311CompletedPhase 3
Novartis Pharmaceuticals
Posted 11/30/2021

A Phase 3 Study of Efficacy and Safety of Remibrutinib in the Treatment of CSU in Adults Inadequately Controlled by H1-antihistamines

NCT05032157CompletedPhase 3
Novartis Pharmaceuticals
Posted 12/1/2021

FDA Halts New Drug Application Acceptance as Government Shutdown Begins

Drug Approval
  • The FDA will not accept new drug applications during the government shutdown, though it can continue reviewing existing submissions funded by user fees.
  • Approximately 86% of FDA staff will remain operational during the shutdown, a higher retention rate than previous shutdowns due to user fee funding.
  • Major pharmaceutical companies including AbbVie, Amgen, Bristol Myers Squibb, J&J and Pfizer could face delays in planned drug submissions.
  • The shutdown compounds existing challenges at the FDA, which has already experienced approval slowdowns due to mass layoffs and leadership changes under the Trump Administration.

Crystalys Therapeutics Emerges with $205M Series A to Advance Gout Treatment Dotinurad

Drug Approval
  • Crystalys Therapeutics launched with $205 million in Series A funding to develop dotinurad, a URAT1 blocker for gout treatment that is already approved in Japan, China, Philippines, and Thailand.
  • The company plans to initiate global Phase III trials for dotinurad in patients who don't respond to initial gout therapies, addressing a significant treatment gap in the second-line space.
  • Dotinurad demonstrated superior uric acid reduction compared to Takeda's Uloric in Phase III studies and offers potential safety advantages over previously withdrawn treatments like Zurampic.

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