TLDR
- uniQure jumps as FDA supports AMT-130 accelerated approval path
- QURE surges after FDA backs Huntington’s gene therapy filing plan
- FDA support gives uniQure a clearer path for AMT-130 approval
- uniQure plans 2026 BLA filing after key FDA regulatory update
- AMT-130 gains FDA filing support as QURE stock sharply rallies
uniQure N.V. (QURE) stock surged 74.29% to $47.04 after the FDA supported a filing path for AMT-130. The stock opened near $27 and climbed sharply before holding near session highs. The move followed new regulatory progress for the Huntington’s disease gene therapy candidate.
FDA Opens Path for AMT-130 BLA Submission
uniQure said the FDA accepted its three-year Phase I/II analysis as the primary basis for a BLA. Therefore, the company plans to seek accelerated approval for AMT-130 in Huntington’s disease. It now expects to submit the application in the third quarter of 2026.
The update followed a recent Type B meeting between uniQure and the FDA. During the meeting, the agency also sought alignment on a confirmatory study design. The company said the FDA will work quickly with it on the remaining requirements.
The confirmatory study may use concurrent controls on standard-of-care therapy instead of a sham procedure. This approach could shape the next stage of the regulatory plan. uniQure expects final meeting minutes within 30 days.
Clinical Data Supports Accelerated Approval Plan
uniQure is testing AMT-130 in multi-center Phase I/II studies across the United States and Europe. The studies assess safety, tolerability, and early efficacy signals in early manifest Huntington’s disease. The program also uses long-term follow-up to track patient outcomes.
The U.S. trial enrolled 26 patients and assigned them to low dose, high dose, or sham procedure groups. Treated patients received a one-time AMT-130 administration through MRI-guided neurosurgical delivery into the striatum. The study includes a 12-month blinded period and five years of follow-up.
The European Phase 1b/2 study enrolled 13 patients with early manifest Huntington’s disease. A third cohort added 12 patients across U.S. and EU sites. Meanwhile, a fourth U.S. cohort is testing high-dose AMT-130 in patients with lower striatal volumes.
Huntington’s Disease Remains a High-Need Treatment Area
Huntington’s disease causes progressive motor, behavioral, and cognitive decline. The inherited disorder results from a CAG repeat expansion in the huntingtin gene. As a result, abnormal protein builds up in the brain and drives worsening symptoms.
Around 75,000 people have Huntington’s disease across the U.S., EU, and UK. Hundreds of thousands more remain at risk of inheriting the condition. However, no approved therapy currently slows disease progression or delays onset.
AMT-130 has received RMAT, Breakthrough Therapy, and Fast Track designations from the FDA. The RMAT status marked the first such designation for Huntington’s disease. Therefore, uniQure now holds several regulatory tools as it prepares its planned BLA filing.
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