uniQure Faces Delay as FDA Walks Back Accelerated Approval for Huntington’s Gene Therapy
uniQure's AMT-130 gene therapy for Huntington’s disease saw a significant regulatory setback after the FDA indicated it is not prepared to review an approval application in its current state, reversing earlier positive signals.137
Earlier in 2025, AMT-130 received Breakthrough Therapy designation from the FDA, and in April, uniQure and the agency were aligned on key elements for accelerated approval.54
Recent mid-stage clinical trial data for AMT-130, showing a 75% slowing in disease progression over three years in a small cohort, had raised investor optimism.1210
Despite prior FDA feedback that seemed positive, a recent meeting led uniQure to believe those results aren't deemed adequate by the FDA for approval, making the timing of a formal submission now unclear.17
uniQure had planned to file for approval in 2026; after the reversal, shares plunged by over 65% as investors reacted to the regulatory uncertainty.1
AMT-130 remains the leading gene therapy in development for Huntington’s disease, but its approval pathway is now significantly delayed as further engagement with the FDA is required.68
Sources:
1. https://www.biopharmadive.com/news/uniqure-fda-huntingtons-gene-therapy-reversal-approval-application/804516/
2. https://www.biopharmadive.com/news/uniqure-huntington-gene-therapy-study-results-fda/760987/
3. https://pharmaphorum.com/news/uniqure-poleaxed-fda-blocks-huntingtons-therapy
5. https://www.uniqure.com/programs-pipeline/huntingtons-disease
6. https://en.hdbuzz.net/additional-clarity-what-we-know-4-weeks-after-the-uniqure-news/
7. https://firstwordpharma.com/story/6514574
8. https://www.biospace.com/drug-development/uniqures-huntingtons-breakthrough-brings-hope-to-patients-but-experts-urge-caution
10. https://www.statnews.com/2025/09/24/huntingtons-gene-therapy-uniqure/