Overview
- uniQure said Wednesday that a formal Type B meeting with the FDA confirmed three‑year follow‑up from its Phase I/II study could serve as the primary data package for an accelerated Biologics License Application.
- The Phase I/II program enrolled 26 U.S. patients and reported that high‑dose recipients showed about a 75% slowing in disease progression at three years on a standard clinical scale.
- The FDA asked uniQure to align on a post‑approval confirmatory study before submission and raised the possibility of using a standard‑of‑care concurrent control instead of sham brain surgery for the control arm.
- Analysts and market data showed an immediate investor reaction, with uniQure shares jumping roughly 70–83% in premarket trading after the agency’s change in stance.
- The decision reverses earlier agency advice that the program’s uncontrolled comparisons were insufficient, a shift that reporting links in part to recent senior FDA leadership changes, and it leaves the company targeting a Q3 2026 BLA filing while final study details are negotiated.