UniQure plans to submit AMT-130 to the U.K.’s Medicines and Healthcare products Regulatory Agency in the third quarter of 2026 based on Phase 1/2 data showing a 75% slowing of disease—the same data the FDA has deemed unacceptable for a biologics license application.
UniQure has a new plan after last year’s FDA imbroglio: head to the U.K. to seek approval of embattled Huntington’s disease gene therapy AMT-130.
UniQure intends to submit the one-time treatment to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) in the third quarter based on the three-year analysis from ongoing Phase 1/2 trials in both the U.S. and Europe, according to a Thursday press release recapping a pre-submission meeting with the regulator. The data—first reported by the biotech in September 2025—showed a 75% slowing of the intractable neurodegenerative disease.
With those data in the vault, uniQure seemed to be on track for a first quarter 2026 submission to the FDA. The U.S. agency foiled those plans, however, telling the company in November that it “no longer agrees” that data from the Phase 1/2 trial are “adequate” to support a BLA submission. This was despite the two parties having previously agreed on the protocols and statistical analyses used. The FDA is now requiring uniQure to conduct a sham surgery–controlled Phase 3 study before it can seek approval of AMT-130.
This does not appear to be the case with the MHRA.
“We are encouraged by the constructive feedback from the MHRA as we continue to work towards a regulatory submission for AMT-130 in Huntington’s disease, which we now expect to submit in the third quarter of this year,” CEO Matt Kapusta said in a statement on Thursday.
“This is an interesting wrinkle in the AMT-130 saga,” Stifel analysts said in a note to investors Thursday morning, adding that uniQure management told the firm the MHRA reached out to the company proactively about AMT-130.
“We suspect QURE may also be benefitting from a vocal KOL community in the UK, where multiple influential KOLs/HD specialists practice,” Stifel continued.
As for the FDA, uniQure revealed that the agency has granted a Type B meeting to take place in the second quarter. At this meeting, the company expects to discuss “key elements of a potential Phase 3 trial design” and receive feedback on the proposed statistical analysis plan for the four-year analysis of the Phase 1/2 trial, which is also expected in the third quarter of this year.
Some industry insiders believe these four-year data, if positive, might still provide an opportunity for earlier approval of AMT-130 in the U.S.
“I’m not convinced that there isn’t a path forward on [upcoming] 48-month data,” Courtney Rice, principal at Acadia Strategy Partners, told BioSpace in March. “I think that the final story has yet to be written here.”
Stifel analysts, however, indicated their belief that the situation in the U.S. remains status quo.
“As we understand it, the idea here is to simply see if FDA has any asks for how these data will be cut/assessed,” they wrote on Thursday. “The current understanding continues to be that the ph1/2 is insufficient for accelerated approval, but it’s nonetheless smart to try to preserve any optionality here in case things change at CBER which they could.”
UniQure is also “actively pursuing additional regulatory pathways in international markets,” for AMT-130, according to the press release, and said it expects to provide additional updates in the second half of 2026.
Editor’s note (April 30): This article has been updated to include commentary from Stifel.
