uniQure (QURE) Submitted AMT-130 for Accelerated Approval in Huntington’s Disease. Can Externally Controlled Data Support the Application?

uniQure has applied to the FDA and UK's MHRA for accelerated approval of AMT-130, a gene therapy for Huntington's disease. The therapy aims to slow disease progression by 75% according to clinical trial data. FDA priority review could shorten the review time to six months.

uniQure submitted a Biologics License Application to the FDA for accelerated approval of its gene therapy AMT-130 for Huntington's disease, with a parallel application to the UK regulator. The therapy, if approved, would be delivered via neurosurgery at specialized centers. The application is based on a Phase 1/2 study with an external control group, and the FDA has not yet decided on priority review.

uniQure (QURE) filed for U.S. and U.K. approval of its Huntington’s disease gene therapy, AMT-130. William Blair resumed coverage with an Outperform rating and $73 price target, citing a 58% upside. Shares initially rose 6% but closed down 3%. The FDA may grant priority review, potentially cutting the review period. AMT-130, if approved, would be uniQure’s second marketed gene therapy.

uniQure (QURE) filed for accelerated approval of its gene therapy AMT-130 for Huntington's disease with the FDA and MHRA. The submissions are backed by three-year Phase 1/2 study data. William Blair resumed coverage with an Outperform rating and $73 target, citing regulatory progress and potential $3.2B peak sales by 2035. QURE shares fell 4.95% on Wednesday.

UniQure's (QURE) stock declined after the FDA accepted its application for a Huntington's gene therapy, resolving a dispute over study requirements. The therapy is now closer to approval.

UniQure has submitted applications to the FDA and UK regulators for approval of its Huntington’s disease gene therapy, AMT-130. The company seeks priority review, which could expedite the FDA's decision to late April or early May 2027. The application is supported by three-year trial data showing a 75% slowing of the disease, despite earlier regulatory challenges. Analysts predict peak global sales of $3.2 billion by 2035, though some regulatory uncertainty remains.