uniQure files BLA and UK MAA for Huntington's gene therapy AMT-130

uniQure N.V. said on September 2, 2026 that it had submitted a Biologics License Application to the U.S. Food and Drug Administration for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for the treatment of Huntington's disease.¹ The company also said its Marketing Authorisation Application for ifezuntirgene inilparvovec has been submitted to the United Kingdom's Medicines and Healthcare products Regulatory Agency.¹

uniQure said it has requested priority review for the BLA, which if granted would shorten the FDA review cycle to six months following the FDA's 60-day BLA filing review period.¹

Both applications rest on the previously announced three-year data analysis from the Phase I/II clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control derived from the Enroll-HD natural history database.¹ uniQure said it intends to present a four-year data analysis from the ongoing Phase I/II clinical studies before the end of the current third quarter.¹

The therapy already carries multiple FDA designations. uniQure said the drug is the first investigational therapy for Huntington's disease to have received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA,¹ and it also holds Fast Track designation from the FDA.¹

On the clinical program, the U.S. study randomized 26 patients, giving 6 the low dose and 10 the high dose in a single administration, while 10 received a sham procedure; four of the sham patients later crossed over to treatment at about 12 months.¹ The open-label European study enrolled 13 patients, 6 at low dose and 7 at high dose.¹ A third group of 12 patients tested both doses combined with immunosuppression, and a fourth group of six U.S. patients is testing the high dose in people with smaller striatal volumes than those in earlier groups.¹

uniQure cautioned that regulators could reach a different conclusion than the company's own reading of the data, noting the possibility that the FDA and MHRA ultimately conclude that the Phase I/II trial data are not sufficient to support regulatory approval, including accelerated approval with respect to a BLA.¹