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FDA Complete Response Letters Highlight Growing CMC Challenges in Immunotherapy Development

Drug Approval
  • Recent FDA complete response letters for Replimune's RP1 and Capricor's CAP-1002 underscore increasing regulatory emphasis on analytical validation, tech transfer execution, and manufacturing controls in immunotherapy development.
  • Despite following FDA guidance throughout development, both companies received unexpected rejections, with 74% of CRLs issued from 2020 to 2024 citing quality or manufacturing deficiencies.
  • Industry experts warn that analytical methods and tech transfer plans that pass early scrutiny often collapse under commercial-scale expectations, requiring CMC rigor to be treated as front-line regulatory strategy.
  • The FDA's heightened scrutiny reflects evolving standards for biologics and advanced therapies, demanding robust manufacturing control strategies and comprehensive CMC data to ensure consistency, safety, and efficacy.

Study of RP1 Monotherapy and RP1 in Combination With Nivolumab

NCT03767348RecruitingPhase 2
Replimune Inc.
Posted 9/20/2017

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT03406780CompletedPhase 2
Capricor Inc.
Posted 4/4/2018

European Regulators Recommend Approval of Vorasidenib for IDH-Mutant Grade 2 Glioma

Drug ApprovalTargeted TherapyOncology
  • The European Medicines Agency's CHMP has issued a positive opinion recommending approval of vorasidenib (Voranigo) for treating grade 2 IDH-mutant glioma in patients aged 12 years and older following surgical resection.
  • The recommendation is based on the phase 3 INDIGO trial showing vorasidenib significantly improved progression-free survival to 27.7 months versus 11.1 months with placebo.
  • If approved by the European Commission, vorasidenib would become the first targeted therapy for grade 2 IDH-mutant diffuse glioma in the European Union.
  • The drug is already approved in the United States, Canada, Australia, Israel, UAE, Saudi Arabia, and Switzerland for this indication.

Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)

NCT04164901Active, Not RecruitingPhase 3
Institut de Recherches Internationales Servier
Posted 1/5/2020

FDA Approves Doptelet for Pediatric Immune Thrombocytopenia with New Sprinkle Formulation

Drug Approval
  • The FDA has approved Doptelet (avatrombopag) for treating thrombocytopenia in pediatric patients one year and older with persistent or chronic immune thrombocytopenia who have had insufficient response to prior therapy.
  • The approval includes a new pediatric-friendly sprinkle formulation for children ages one to less than six years, offering flexible oral administration with no food restrictions.
  • In the pivotal AVA-PED-301 phase 3 study, 27.8% of patients receiving Doptelet achieved durable platelet response compared to 0% in the placebo group, with 81.5% achieving alternative platelet response endpoints.
  • The oral thrombopoietin receptor agonist was generally well-tolerated, with the most common side effects being viral infections, nasopharyngitis, cough, fever, and sore throat.

Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months

NCT04516967Active, Not RecruitingPhase 3
Sobi, Inc.
Posted 3/5/2021

EU Regulatory Approval Advances Pfizer-BioNTech LP.8.1-Adapted COVID-19 Vaccine for 2025-2026 Season

Drug Approval
  • The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended approval for Pfizer and BioNTech's LP.8.1-adapted COMIRNATY vaccine targeting XFG and NB.1.8.1 variants.
  • The updated vaccine demonstrates improved immune responses against emerging SARS-CoV-2 sublineages and maintains a safety profile consistent with prior formulations.
  • European Commission authorization is expected to enable vaccine deployment as early as fall 2025 for the 2025-2026 season, with manufacturing already underway at risk.
  • The global mRNA vaccines and therapeutics market is projected to grow at 13.3% CAGR, expanding from $53.14 billion in 2025 to $144.31 billion by 2033.

Insmed's Brensocatib Wins FDA Approval as First Treatment for Non-Cystic Fibrosis Bronchiectasis

Drug ApprovalRare DiseaseOrphan Drug
  • The FDA approved Insmed's brensocatib (Brinsupri) as the first treatment for non-cystic fibrosis bronchiectasis, a progressive lung condition that can cause permanent lung damage.
  • The approval represents a "best-case scenario" with both doses approved and no major warnings added, positioning the drug for a potentially rapid commercial launch.
  • Analysts project Brinsupri could generate $3.8 billion in U.S. sales and over $6 billion worldwide, with the drug priced at $88,000 annually.
  • Insmed is also developing brensocatib for additional inflammatory conditions including chronic rhinosinusitis and hidradenitis suppurativa, leveraging its DPP1 enzyme blocking mechanism.

FDA Grants Priority Review for Flibanserin Expansion to Postmenopausal Women with HSDD

Drug Approval
  • The FDA has granted Priority Review to a supplemental new drug application seeking to expand flibanserin 100 mg (Addyi) indication to include postmenopausal women with hypoactive sexual desire disorder.
  • Flibanserin is currently the only FDA-approved, non-hormonal oral therapy for low sexual desire in premenopausal women, and this expansion would address an unmet need for postmenopausal women.
  • The application is supported by robust clinical trial data from the largest clinical trials in women's sexual health, with Health Canada already approving the drug for postmenopausal women based on the same evidence.
  • If approved, this would represent the first FDA-approved pharmacologic therapy for HSDD in postmenopausal women, potentially benefiting the estimated 40% of women who experience low sexual desire at some point in their lives.

Flibanserin for the Treatment of Hypoactive Sexual Desire Disorder in Postmenopausal Women in North America

NCT01057901TerminatedPhase 3
Sprout Pharmaceuticals, Inc
Posted 1/1/2010

Incyte Reports Strong Q2 2025 Results with Jakafi Growth and New Drug Approvals

Drug ApprovalMonoclonal AntibodyOncology
  • Incyte reported strong second quarter 2025 financial results with Jakafi net revenues of $764 million, representing 8% year-over-year growth driven by increased demand across all indications.
  • The company achieved two significant regulatory milestones with FDA approvals of Zynyz for squamous cell anal carcinoma and Monjuvi for follicular lymphoma, expanding treatment options for cancer patients.
  • Niktimvo, launched in Q1 2025, demonstrated continued strong commercial uptake with $13.6 million in sales, while Opzelura revenues grew 35% to $164 million driven by increased patient demand in both atopic dermatitis and vitiligo.
  • The company raised its 2025 revenue guidance for Jakafi and other oncology products, reflecting strong first-half performance and successful product launches.

MAXPIRe: Study to Evaluate Axatilimab in Participants With Idiopathic Pulmonary Fibrosis (IPF)

NCT06132256RecruitingPhase 2
Syndax Pharmaceuticals
Posted 12/11/2023

A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease

NCT06585774RecruitingPhase 3
Incyte Corporation
Posted 1/21/2025

A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

NCT06388564RecruitingPhase 2
Incyte Corporation
Posted 10/11/2024

Eisai Launches Beova Tablets for Overactive Bladder Treatment in Thailand

Drug Approval
  • Eisai Thailand has launched Beova® Tablets (vibegron), a selective β3-adrenergic receptor agonist for overactive bladder treatment, marking the first market entry in Eisai's licensed ASEAN region.
  • The once-daily oral medication targets β3 receptors in the bladder to improve symptoms of urgency, frequent urination, and urge incontinence in patients with overactive bladder.
  • With overactive bladder affecting 15.8% of Thailand's population, the launch provides a new treatment option alongside Eisai's existing Uritos® tablets for addressing this condition that significantly impacts quality of life.
  • Approvals have also been obtained in the Philippines and Malaysia, with market preparations underway for these territories following the successful Thailand launch.

FDA Approves First 3D-Printed Epilepsy Drug for Feeding Tube Administration

Drug Approval
  • The FDA has approved Spritam (levetiracetam), the world's first 3D-printed medication, for administration via nasogastric and gastrostomy tubes, making it the only levetiracetam formulation with this approval.
  • The drug uses Aprecia's proprietary ZipDose Technology Platform to create rapidly disintegrating tablets that can be dispersed in minimal liquid volumes for enteral administration.
  • This approval provides critical access for epilepsy patients who cannot take oral medications, particularly those with swallowing difficulties or requiring enteral feeding support.
  • Spritam is indicated for treating partial-onset seizures in patients 4 years and older, myoclonic seizures in juvenile myoclonic epilepsy patients 12 years and older, and generalized tonic-clonic seizures in patients 6 years and older.

CDSCO Approves Phase III Trials for Two Novel Cardiovascular Fixed-Dose Combinations

Drug Approval
  • India's CDSCO has approved Phase III clinical trials for two innovative fixed-dose combinations targeting cardiovascular conditions, including Ravenbhel's dapagliflozin-sacubitril-valsartan triple combination and Zydus Healthcare's empagliflozin-metoprolol dual combination.
  • Ravenbhel received approval to conduct both bioequivalence studies and Phase III trials for their triple FDC, which combines an SGLT2 inhibitor with a neprilysin inhibitor and ARB for comprehensive heart failure management.
  • Zydus Healthcare secured approval for Phase III trials of their empagliflozin-metoprolol combination after submitting a revised protocol, targeting improved patient compliance in cardiovascular care.
  • Both approvals represent significant regulatory milestones for advancing combination therapies that could simplify treatment regimens for patients with complex cardiovascular conditions.

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