uniQure submits long-awaited BLA for Huntington's gene therapy

uniQure has submitted its BLA to the U.S. FDA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for the treatment of Huntington’s disease.

The company has requested priority review for the BLA, which, if granted, would shorten the review cycle to six months following the FDA’s 60-day BLA filing review period. uniQure also announced that its marketing authorization application for AMT-130 has been submitted to the UK’s MHRA.

The BLA and MAA are supported by the previously announced three-year data analysis from the phase 1/2 clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control derived from the Enroll-HD natural history database. uniQure intends to present a four-year data analysis from the ongoing phase 1/2 clinical studies before the end of the current third quarter.

The FDA submission comes after much back and forth between uniQure and the agency. The trouble started during uniQure’s pre-BLA meeting with the FDA in October 2025, when the agency communicated that it didn’t believe that the data submitted from the phase 1/2 studies of AMT-130 provided the primary evidence to support a BLA submission. According to uniQure, that was a shift from prior communications with the FDA.

In February 2026, FDA Commissioner Marty Makary appeared on CNBC and made comments that appeared to reference and disparage AMT-130. “There was a product where the researchers drilled a burr hole, literally a hole, in people’s skulls,” said Makary. “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.”

Then, in March, final Type A meeting minutes from the FDA confirmed tthe agency's stance that the data was insufficient to support a marketing application. The minutes from the meeting also strongly recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study — a problematic suggestion given that AMT-130 is administered as a one-time gene therapy directly into the brain via a complex neurosurgical procedure.

Following Dr. Vinay Prasad's resignation from CBER in March and Makary's resignation in May the agency reversed course in June, deciding that that the 3-year analysis from the phase 1/2 study would be acceptable as the primary basis of a BLA. 

AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging the company’s proprietary miQURE silencing technology. The therapeutic goal is to inhibit the production of the mutant protein. If approved, it will be the first genetic treatment for Huntington’s disease.