After a year of controversial regulatory reversals, the FDA has accepted uniQure’s application for ifezuntirgene inilparvovec for priority review, with a May 1 action date. If approved, the therapy—also called AMT-130—would be the first disease-modifying treatment for Huntington’s disease.
At long last, the FDA has accepted uniQure’s embattled Huntington’s disease gene therapy for priority review, setting a May 1 decision date for the product, with a potential advisory committee meeting in the works.
Despite the investigational gene therapy showing a 75% slowing of disease after 36 months in a Phase 1/2 trial in September 2025, FDA officials at the time took issue with the design of the study and insisted that uniQure run a sham-controlled Phase 3 study before submitting an application for approval. After a reversal of guidance this spring, the biotech filed for approval of ifezuntirgene inilparvovec—also called AMT-130—on Sept. 2.
If approved, ifezuntirgene inilparvovec would be the first disease-modifying treatment for Huntington’s, a rare fatal, inherited neurodegenerative disorder that causes motor symptoms, behavioral abnormalities and cognitive decline.
Stifel analysts saw the biologics license application (BLA) acceptance as a positive sign for the therapy and biotech. “We think the BLA acceptance today, with priority review, represents an incrementally positive sign on the prospects of accelerated approval,” the group wrote in an investor note on Monday morning.
William Blair agreed with Stifel’s assessment. “We view this announcement as an incremental positive given that it supports the FDA’s willingness to review the application for accelerated approval,” the firm said in a Monday note to investors.
UniQure is anticipating that the FDA will call an advisory committee meeting for the application, since the agency stated that it is “tentatively planning to discuss this application” at an adcomm, the company said in a Monday morning press release.
UniQure has walked a rocky path to get to this moment. In November 2025—five weeks after revealing the three-year data that sparked excitement and optimism in the Huntington’s community—the FDA reversed previous guidance that such data would be sufficient to support an approval application. Instead, the agency said the data were not “adequate to provide the primary evidence in support of a BLA submission,” according to uniQure at the time.
In the following months, then FDA Commissioner Marty Makary and Center for Biologics and Evaluation (CBER) director Vinay Prasad proceeded to make negative comments appearing to allude to the gene therapy. Both leaders have since left the agency.
Then in September this year, four-year data from the Phase 1/2 trial in were “weaker than expected,” Stifel wrote in Monday’s note, causing uniQure’s stock to drop by as much as 38%. The analysts noted that the company has traded down since.
In a statement on Monday, uniQure CEO Matt Kapusta called the BLA acceptance “an important milestone for the Huntington’s disease community, which has waited generations for a treatment that changes the course of this devastating disease.”
UniQure also provided an update Monday on the asset’s confirmatory trial, saying this “remains a key priority, with screening anticipated to begin by year end.” The company expects the study to be “well underway by the time of potential approval.”
The study will enroll around 200 patients randomized to receive ifezuntirgene inilparvovec or standard of care. The main goal will be total functional capacity—a primary scale used in Huntington’s to measure a patient’s ability to complete every-day tasks—over 36 months.
