UniQure files for FDA, UK approval of first gene therapy for Huntington’s disease

News FDA UniQure files for FDA, UK approval of first gene therapy for Huntington’s disease September 2, 2026 | 3 min read | Heather McKenzie Twitter LinkedIn Facebook Email Print The biologics license application follows months of public speculation—and regulatory reversals—for uniQure’s AMT-130. If accepted by the FDA for priority review, approval could come in the second quarter of 2027. After months of regulatory spectacle, uniQure has submitted a biologics license application to the FDA for its Huntington’s disease gene therapy.

The biotech has also applied for approval of AMT-130 in the United Kingdom.“We are grateful to the FDA for its leadership in advancing regulatory science to meet the urgency of this disease, and to the MHRA [Medicines and Healthcare products Regulatory Agency] for its commitment to advancing rare disease treatments in the U.K.,” uniQure CEO Matt Kapusta said in a statement on Wednesday. “We look forward to working with both agencies as these applications progress.”The biotech has requested FDA priority review for the BLA submission. If granted, this would mean a six-month review cycle for AMT-130, following a 60-day BLA review period, uniQure noted in the press release.

If accepted by the agency for priority review, FDA could set an action date for the potential accelerated approval in late April or early May 2027—depending on the exact date of the submission.If approved, AMT-130 would be the first-ever genetic treatment for Huntington’s disease.UniQure is backing its application with three-year data from a Phase 1/2 trial that compared AMT-130 to a propensity score-matched external control derived from the Enroll-HD natural history database. The data, announced in September 2025, showed a 75% slowing of the intractable neurodegenerative disease. Based on these results, the company was all set to seek approval of the gene therapy in the first quarter of this year.However, uniQure ran into regulatory roadblocks under former FDA Commissioner Marty Makary and former Center for Biologics Evaluation and Research (CBER) director Vinay Prasad, who both spoke out publicly against the therapy.In November 2025, the FDA told uniQure it “no longer agrees” that data from the company’s Phase 1/2 trial are “adequate to provide the primary evidence in support of a BLA submission,” per a uniQure update at the time.

The two parties had previously agreed on the protocols and statistical analyses used, according to the biotech.In February, Makary appeared to disparage AMT-130 in an interview with CNBC. “There was a product where the researchers drilled a burr hole, literally a hole, in people’s skulls” to directly inject a drug candidate into patients’ brains, Makary said during the interview. “At the end of the randomization period, it was found no benefit, and yet this is one of the drugs that we were pressured to approve.” While Makary did not name the product, it was widely believed that he was referring to uniQure’s gene therapy.Four months later, Makary and Prasad were both gone, and the FDA again reversed course on AMT-130, agreeing that the three-year data were sufficient to support an application for accelerated approval.

Huntington’s disease UniQure plans Q3 submission for Huntington’s gene therapy after FDA reverses course—again After a regulatory odyssey that delayed a filing for what would be the first genetic medicine for Huntington’s disease, the FDA has agreed that three-year data from uniQure’s Phase 1/2 trial are sufficient to support an accelerated biologics license application. June 17, 2026 · 4 min read · Heather McKenzie Read more Even though the two leaders have left the agency, analysts still see some uncertainty ahead for uniQure.“We recognize some regulatory risk remains given the continued leadership changes at the FDA,” William Blair wrote in a Wednesday morning note to investors announcing resumption of coverage for the stock. The firm also highlighted that the Phase 1/2 trial design “aligns with the FDA’s plausible mechanism pathway publication and draft guidance on gene therapies for small populations, which specifically stipulates that external controls may be an appropriate option in certain contexts.”In a follow-up note, William Blair added that alignment has been reached between uniQure and the FDA on key elements of a confirmatory trial.

“In a follow-up with management, it indicated that the company reached alignment on the key elements of the confirmatory trial.” the analyst said.This alignment “is another positive sign” for uniQure, Stifel agreed in its own Wednesday note.The company is “confident” that the study—which will include around 200 patients with 2:1 randomization between AMT-130 and standard of care— “will be well underway before the FDA gives their decision on accelerated approval, which is in-line with the agency’s expectations,” the analysts wrote.BioSpace has reached out to uniQure for further information on the confirmatory trial. William Blair also predicted a “measured” launch for AMT-130 “given the requirement for surgically guided administration.” The analyst estimated peak global sales of $3.2 billion in 2035.“Given the impressive results demonstrated with the therapy, we believe AMT-130 has the potential to be a first- and best-in-class treatment for HD,” William Blair said. Twitter LinkedIn Facebook Email Print Huntington’s disease FDA Regulatory uniQure Heather McKenzie Heather McKenzie is senior editor at BioSpace and curator of the ClinicaSpace newsletter.

She is an award-winning journalist specializing in rare disease and neuroscience, in addition to her extensive coverage of the FDA and regulatory science. You can reach her at heather.mckenzie@biospace.com. Also follow her on LinkedIn.