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NICE Recommends Vanzacaftor-Tezacaftor-Deutivacaftor for Cystic Fibrosis Treatment in England

Drug Approval
  • NICE has approved vanzacaftor-tezacaftor-deutivacaftor (Alyftrek) for NHS funding in England to treat cystic fibrosis in patients aged 6 years and older with at least one F508del mutation.
  • Clinical trial evidence demonstrates the triple combination therapy is as effective as the current standard treatment ivacaftor-tezacaftor-elexacaftor in improving lung function, growth, weight gain, and reducing lung infections.
  • The new therapy must be funded within 30 days of final guidance publication, with cost comparisons suggesting similar expenses to existing treatments.
  • NICE determined there is sufficient evidence showing the treatment provides benefits and value for money, allowing routine use across the NHS for this patient population.

AscellaHealth and Abeona Therapeutics Launch Comprehensive Support Program for ZEVASKYN Gene Therapy

Rare DiseaseDrug Approval
  • AscellaHealth has partnered with Abeona Therapeutics to successfully commercialize ZEVASKYN, the first FDA-approved autologous cell sheet-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB).
  • The collaboration resulted in AbeonaAssist, a customized patient support program designed to address the unique clinical, operational, and reimbursement challenges of this groundbreaking cell-based gene therapy.
  • Early results demonstrate enhanced patient compliance, retention, and satisfaction rates, highlighting the effectiveness of tailored support programs for complex gene therapies.
  • ZEVASKYN incorporates the functional COL7A1 gene into patients' own skin cells to produce type VII collagen, demonstrating clinically meaningful wound healing and pain reduction with a single surgical application.

China Medical System Secures NMPA Acceptance for ZUNVYL Alzheimer's Drug Application

Drug Approval
  • China Medical System Holdings announced that China's National Medical Products Administration has accepted the new drug application for ZUNVYL, a treatment for mild-to-moderate Alzheimer's dementia.
  • ZUNVYL is a next-generation acetylcholinesterase inhibitor that demonstrated gastrointestinal adverse events of less than 2% across all studies, potentially addressing tolerability issues that cause 55% of patients to discontinue medication after one year.
  • The drug was previously approved by the FDA in July 2024 and represents only the second oral therapy approved for Alzheimer's disease treatment in the past decade.
  • China has approximately 9.83 million patients with Alzheimer's dementia, with 7.93 million classified as mild-to-moderate cases, highlighting significant market potential for improved treatment options.

Ivonescimab Demonstrates Superior Efficacy in Phase III Lung Cancer Trials, Positioning for Global Regulatory Approvals

Drug Approval
  • Ivonescimab, a PD-1/VEGF bispecific antibody, achieved a 48% reduction in progression-free survival risk compared to chemotherapy in EGFR-mutated NSCLC patients.
  • The drug outperformed pembrolizumab in head-to-head comparison with median PFS of 11.14 months versus 5.82 months in PD-L1-positive NSCLC.
  • Akeso has secured two NMPA approvals in China for 2025, while Summit Therapeutics prepares BLA filing for U.S. market entry.
  • The projected $500 million market opportunity reflects growing adoption across Asia-Pacific region with 15% CAGR expected through 2033.

China Approves XPOVIO for Second-Line Multiple Myeloma Treatment Based on Phase III BENCH Trial

Drug Approval
  • China's NMPA has approved XPOVIO (selinexor) in combination with bortezomib and dexamethasone for adult multiple myeloma patients who have received at least one prior therapy.
  • The approval is based on the Phase III BENCH trial involving 154 Chinese patients, which demonstrated superior efficacy compared to standard bortezomib-dexamethasone treatment.
  • XPOVIO is the world's first approved orally-available selective XPO1 inhibitor and marks the third approved indication for the drug in China.
  • The drug has already been approved in ten Asia-Pacific countries and included in national insurance schemes in five markets.

Biogen's Zurzuvae Receives EMA Panel Recommendation for Postpartum Depression Treatment

Drug Approval
  • The European Medicines Agency's Committee for Medicinal Products for Human Use has recommended marketing authorization for Biogen's ZURZUVAE (zuranolone) for treating postpartum depression in adults following childbirth.
  • The recommendation is based on the SKYLARK study, where ZURZUVAE demonstrated significant reduction in depressive symptoms as early as Day 3 and sustained through Day 45 compared to placebo.
  • If approved by the European Commission in Q3 2025, ZURZUVAE would become the first treatment specifically authorized within the European Union for postpartum depression.
  • The drug is a neuroactive steroid that enhances GABA activity and will be available as 20 mg, 25 mg, and 30 mg hard capsules.

A Study to Evaluate the Efficacy and Safety of SAGE-217 in Participants With Severe Postpartum Depression (PPD)

NCT04442503CompletedPhase 3
Biogen
Posted 6/8/2020

Health Canada Approves Clearside Biomedical's XIPERE for Uveitic Macular Edema Treatment

Drug Approval
  • Health Canada has granted approval for XIPERE (triamcinolone acetonide injectable suspension) for suprachoroidal use in treating uveitic macular edema, expanding the drug's global reach.
  • XIPERE is now approved in the United States, Canada, Australia, and Singapore, with regulatory review ongoing in China, demonstrating continued international validation.
  • The approval utilizes Clearside's proprietary suprachoroidal space injection platform featuring the SCS Microinjector, which provides targeted drug delivery to the back of the eye.
  • Bausch + Lomb holds exclusive commercialization rights for XIPERE in the US and Canada, while Arctic Vision has licensing rights across Greater China and other Asian markets.

EMA Recommends Approval of Aqneursa for Niemann-Pick Type C Disease Treatment

Drug ApprovalRare DiseaseOrphan Drug
  • The European Medicines Agency's Committee for Medicinal Products for Human Use has issued a positive opinion recommending approval of Aqneursa (levacetylleucine) for treating neurological manifestations of Niemann-Pick type C disease in adults and children.
  • The recommendation is based on Phase III trial data showing statistically significant improvement in neurological symptoms within 12 weeks, with patients demonstrating better SARA scores compared to placebo.
  • Aqneursa represents a potential new frontline treatment option for NPC patients in Europe, following its FDA approval in September 2024.
  • The drug targets underlying neurological dysfunction processes and can be used in combination with miglustat or as monotherapy when miglustat is not tolerated.

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288RecruitingPhase 3
IntraBio Inc
Posted 6/30/2022

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.

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

NCT03406780CompletedPhase 2
Capricor Inc.
Posted 4/4/2018

Study of RP1 Monotherapy and RP1 in Combination With Nivolumab

NCT03767348RecruitingPhase 2
Replimune Inc.
Posted 9/20/2017

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

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