FDA grants uniQure’s Huntington’s treatment priority review, sets May 1, 2027 decision date
Key Facts
uniQure said the U.S. Food and Drug Administration accepted its biologics license application for the experimental treatment AMT-130 and granted it priority review, setting May 1, 2027, as the target action date. The date sets a timetable for deciding on the application under the Prescription Drug User Fee Act, but does not mean the treatment has been approved. The FDA says priority review aims for action within 6 months of receiving an application, compared with 10 months for standard review. The agency said the designation accelerates the target review timeline but does not change the scientific or medical standard for approval. The regulatory decision comes as the treatment’s efficacy and safety remain subject to the agency’s review of the filing.
The application was based on 36-month data from 12 patients who received the high dose, according to uniQure’s disclosure to the U.S. Securities and Exchange Commission. The company said the FDA told it at a Type B meeting in June 2026 that the data would be acceptable as the primary basis for the application. The company had submitted the application before announcing additional topline results on September 29, 2026. It said in its disclosure that those results were not part of the application submitted to the agency. The data underpinning the application therefore predate the clinical update that later raised questions about the trial results at longer follow-up.
The additional results concerned the cUHDRS measure at 48 months and did not reach statistical significance in showing a slowing of disease progression, according to a Reuters report published on September 29. Reuters reported that uniQure’s U.S.-listed shares fell 40% that day after the results were announced. The reaction highlights the sensitivity of the company’s valuation to trial data, as the application review proceeds on the basis of the earlier dataset the company identified with the FDA. Priority review is not a judgment on the later results or a waiver of the evidentiary standard required for approval. The agency must evaluate the filing under its standards before the target action date in May 2027.
uniQure described AMT-130 as an experimental gene therapy administered once through an MRI-guided surgical procedure targeting the brain’s striatum. The company said the treatment uses a gene-silencing platform targeting the huntingtin gene. According to the company’s disclosure, there are no approved treatments to delay the onset of Huntington’s disease or slow its progression. If approved, the treatment would represent a new therapeutic approach for the disease, but the company has presented it as an experimental candidate, not an approved treatment. The current step is acceptance and review of the application, with an FDA decision due by the announced target date.
QURE shares closed at $24.83 on October 30, 2026, according to EL7 data. The price follows the decline Reuters reported after the September update, while data for the session when the application’s acceptance was announced on November 2 are not available here. uniQure is scheduled to report its third-quarter 2026 financial results on November 4, 2026, according to the company’s investor and media calendar. That is about six months before the FDA’s target date, under the announced priority review timeline. The agency’s decision on the AMT-130 application will be the next regulatory milestone identified in the available evidence.