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In another reversal, FDA gives uniQure accelerated approval path for Huntington's gene therapy

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The FDA has reversed its earlier stance and will allow uniQure to pursue accelerated approval for its Huntington's disease gene therapy, AMT-130, based on a three‑year analysis from its Phase I/II study compared to an external control. UniQure plans to submit a Biologics License Application (BLA) in the third quarter of 2026 for AMT‑130, avoiding the need to conduct a new Phase III trial before filing. The FDA agreed that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) can serve as an intermediate clinical endpoint for accelerated approval, with reductions in neurofilament light chain (NfL) in cerebrospinal fluid as supportive evidence. The agency also agreed to align on a confirmatory study design prior to BLA submission, including consideration of a concurrent control on standard‑of‑care therapy instead of a sham procedure, and expressed commitment to working expeditiously with uniQure. AMT‑130 already holds Regenerative Medicine Advanced Therapy (RMAT), ...

Moderna adds commercial chief, expands Stephen Hoge's role

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Alvotech Announces Closing of Public Offering and Full Exercise by Underwriters of Option to Purchase Additional Shares, Raising an Aggregate of $165 Million with Concurrent Private Placement

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Moderna Eyes German Vaccine Facilities as Senate Democrats Push Broad Drug Pricing Reforms

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After FDA sign-off, Colorado's drug import plan faces tough road ahead

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Novo Nordisk hit by multi‑million‑dollar ransom demands after major data breach claimed by hacking groups

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UniQure stock surges after FDA U-turn clears path to gene therapy filing

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Despite 'decent' data, Verastem rethinks options for approved oncology combo in pancreatic cancer

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Jazz partners with AbCellera on T-cell engager programs for GI cancers in deal valued up to $4.1B, with $56M upfront and up to $792M in milestones per program, plus tiered royalties on net sales spanning mid-single-digit to low double-digit percentages.

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Merck adds to biopharma’s AI agenda with up to $510M+ Protillion pact

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Lilly bolsters non‑opioid pain portfolio with 4E Therapeutics buy

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Migraine biotech makes its debut with $46M to advance preclinical pipeline

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Oncolytics Biotech Secures New U.S. Patent Protecting Commercial Manufacturing of Pelareorep into 2044

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