FDA accepts uniQure Huntington’s disease gene therapy BLA and grants priority review
uniQure N.V. announced that the U.S. Food and Drug Administration has accepted its Biologics License Application for ifezuntirgene inilparvovec (AMT‑130) and placed it under priority review. The agency set a PDUFA target action date of May 1 2027 and indicated a tentative advisory‑committee meeting. The company plans a confirmatory trial enrolling about 200 patients, with U.S. screening to begin by the end of 2026.
Why it matters
If approved, the therapy would be the first disease‑modifying treatment for Huntington’s disease, a market with no existing options. The regulatory milestone also drives share‑price volatility, as investors reassess the drug’s commercial potential and the timeline for potential revenue.
Key facts
- 1The FDA accepted uniQure’s BLA for ifezuntirgene inilparvovec (AMT‑130) and granted priority review. stocktitan.net
- 2The PDUFA target action date is May 1 2027. stocktitan.net
- 3The therapy holds Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations. stocktitan.net
- 4uniQure plans a randomized confirmatory trial with approximately 200 patients (2:1 treatment to standard‑of‑care). stocktitan.net
- 5Patient screening for the confirmatory trial is expected to begin by the end of 2026. stocktitan.net
- 6Shares fell 7.49% to $22.97 after the announcement. rttnews.com
Open questions
- Some reports described a pre‑market share rise of 2‑6%, while others noted a post‑announcement decline of 5‑7%.
Summary written by AlphAI AI Desk from 14 of 14 sources. Not investment advice. Figures are as stated by the linked sources.