FDA Accepts uniQure's Huntington's Gene Therapy Filing With a May 1 Decision Date
- The FDA has agreed to review uniQure's application for its Huntington's disease gene therapy on an accelerated timeline, with a decision due May 1.
- The agency also tentatively plans to consult external advisers on the application.
- Shares fell about 7% by late Monday, though analysts mostly read the update as positive; TD Cowen models annual sales reaching $1.6 billion by 2039.
💡 Points Based on public reports
A first approval in Huntington's would be a landmark for CNS gene therapy, but the open question is the evidence package: a committee meeting signals the agency wants outside views on the data and on how benefit is judged in a slowly progressing disease. The stock dipping on good news suggests investors had priced a faster path. Compare how regulators treated other single-arm gene therapy filings before assuming approval.
UniQure gets an FDA decision date; Teva scraps brain drug study ↗