NEW YORK, June 18, 2026, 06:04 EDT
- uniQure shares jumped 78.4% to finish Wednesday at $48.16 after the FDA said data from the main AMT-130 study could back an accelerated-approval filing.
- uniQure said it will file a Biologics License Application to the FDA in the third quarter of 2026, seeking approval to market its biological medicine in the U.S.
- The move comes after a period of regulatory swings. The FDA still needs to approve a confirmatory trial and review the full filing.
uniQure N.V. shares on the Nasdaq held much of Wednesday’s big jump in premarket trade Thursday. The U.S. FDA has cleared a route for the Netherlands-based gene-therapy firm to pursue approval of its experimental Huntington’s disease drug, AMT-130. The stock finished at $48.16 on Wednesday, climbing 78.4% from $26.99 a day before.
uniQure says the news is key right now since AMT-130 is its main pipeline asset and there’s no approved drug for Huntington’s disease that puts off symptoms or slows things down. Huntington’s is a genetic brain disease causing movement problems, mental decline, and changes in behavior.
uniQure said the FDA told the company it can use a three-year update from its Phase I/II trial as the foundation for a Biologics License Application through the accelerated approval route. This pathway lets the FDA clear drugs for serious illnesses if data is seen as likely to predict a benefit, though more evidence has to come later. uniQure plans to file in the third quarter.
The FDA told uniQure its clinical data is enough for a near-term BLA filing, Chief Executive Matt Kapusta said in the company’s statement. Kapusta also said the agency will help uniQure set up the needed confirmatory trial.
This is a shift from earlier this year. Reuters said the FDA had pushed uniQure to do another trial that would have required a sham surgery, which involves a fake operation. That raised ethical concerns since AMT-130 goes straight into the brain. Now, according to uniQure, the FDA is looking at a control group on standard-of-care therapy instead.
Data for the new filing plan are limited. uniQure said it enrolled 26 patients with early manifest Huntington’s disease in its U.S. Phase I/II trial, and the European open-label study had 13. Reuters said last year’s data pointed to a 75% drop in disease progression for high-dose AMT-130 patients.
Analysts said the move signals the FDA is easing up on rare-disease cases. Guggenheim’s Debjit Chattopadhyay pointed to better understanding of how hard it is to run rare-disease studies. William Blair’s Myles Minter said the regulator looks “more flexible on regulatory paths” when concerns come up. Reuters
PTC Therapeutics and Novartis are in the race too. Their oral Huntington’s drug votoplam is moving forward. In April, PTC said 24-month extension data showed a 52% slowdown in disease progression for stage 2 patients at 10 mg versus a matched group. Novartis has started a global Phase 3 trial aimed at enrolling about 770 patients.
FDA action is still the key for the rally. uniQure expects final minutes from its meeting in the next 30 days, but its risk disclosure warns the FDA may judge Phase I/II data as insufficient for a filing or accelerated approval. Additional patient data could also affect how the three-year results look.
U.S. markets hadn’t opened yet in New York. Nasdaq’s 2026 schedule puts a market holiday on Friday, June 19, for Juneteenth. That means Thursday is the last normal trading day before the extended weekend.
