uniQure N.V. QURE
uniQure N.V. (Nasdaq: QURE) is a CNS/neurology/psychiatry drug developer. Its lead program, ifezuntirgene inilparvovec (AMT-130), is in Phase 3 for Huntington's disease. Its next catalyst on record is ifezuntirgene inilparvovec (AMT-130): Confirmatory study initiation (December 2026).
Upcoming catalysts
- December 2026ifezuntirgene inilparvovec (AMT-130) — Confirmatory study initiation
We plan to initiate patient screening in the United States by the end of 2026.
- May 2027ifezuntirgene inilparvovec (AMT-130) — PDUFA target action date
The application has been granted Priority Review with a Prescription Drug User Fee Act ("PDUFA") target action date of May 1, 2027.
Next further out: ifezuntirgene inilparvovec (AMT-130) — FDA Advisory Committee meeting (date not stated). 1 catalyst on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| ifezuntirgene inilparvovec (AMT-130) | Huntington's disease | gene therapy | Phase 3 |
| AMT-260 | refractory mesial temporal lobe epilepsy (MTLE) | gene therapy | Phase 1 |
| AMT-191 | Fabry disease | gene therapy | Phase 1 |
| AMT-162 | Amyotrophic lateral sclerosis caused by mutations in SOD1 | gene therapy | Phase 1 |
Recent results and decisions
- Nov 2, 2026accepted FDA accepts and grants Priority Review to the BLA for ifezuntirgene inilparvovec for Huntington's disease with a PDUFA target action date of May 1, 2027.
The application has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of May 1, 2027.
Filing - Nov 2, 2026started Randomized confirmatory trial of approximately 200 patients with Total Functional Capacity as primary endpoint at 36 months to start patient screening by year-end 2026.
The study is designed as a randomized, standard-of-care trial enrolling approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with Total Functional Capacity (TFC) as the primary endpoint at 36 months.
Filing - accepted FDA granted priority review of BLA for ifezuntirgene inilparvovec with PDUFA target action date of May 1, 2027.
In November 2026, the U.S. Food and Drug Administration (FDA) accepted for review the Company's Biologics License Application (BLA) for ifezuntirgene inilparvovec, submitted under the Accelerated Approval pathway. The BLA was granted Priority Review with a Prescription Drug User Fee Act (PDUFA) targ
Filing
8 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 CNS/neurology/psychiatry companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.