UniQure submits FDA and UK applications for first Huntington’s disease gene therapy
UniQure has filed a biologics license application (BLA) with the U.S. FDA and a separate submission with the UK Medicines and Healthcare products Regulatory Agency (MHRA) for its experimental gene therapy AMT-130 aimed at treating Huntington’s disease.
The company is seeking priority review from the FDA, which would shorten the review timeline to roughly six months after an initial 60‑day filing assessment. If granted, the agency could set an action date for a potential accelerated approval in late April or early May 2027.
Approval of AMT-130 would represent the first genetic treatment for Huntington’s disease, a rare neurodegenerative condition with no disease‑modifying therapies currently available. UniQure’s CEO Matt Kapusta thanked the regulators for their support and said the company looks forward to working with both agencies as the applications move forward.
This writeup was produced by pharmadog from original reporting by BioSpace.
Original headline: “UniQure files for FDA, UK approval of first gene therapy for Huntington’s disease”
read at BioSpace ↗
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