FDA reverses course on UniQure gene therapy for Huntington's

uniQure announced that, during a recent Type B meeting with the U.S. FDA, the agency decided that that the 3-year analysis from the company’s phase 1/2 study would be acceptable as the primary basis of a BLA for the accelerated approval of AMT-130 in Huntington’s disease.

In addition, the FDA seeks to align on the confirmatory study design prior to the BLA submission, including consideration of concurrent control on standard-of-care therapy instead of a sham procedure. The suggestion of a sham procedure was a point of conflict between uniQure and the FDA, given that AMT-130 is administered as a one-time gene therapy directly into the brain via a single, complex neurosurgical procedure.

The alignment with the FDA hopefully marks the end of much back and forth between uniQure and the agency. Back in March, uniQure announced that it had received final Type A meeting minutes from the FDA regarding AMT-130, with the agency stating that data from the phase 1/2 studies are not sufficient to support a marketing application. The minutes from the meeting strongly recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study — feedback that uniQure said was a shift from prior communications with the FDA in multiple Type B meetings over the past year.

Now, uniQure intends to submit its BLA in the third quarter of 2026.

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. AMT-130 has been granted breakthrough therapy designation and regenerative medicine advanced therapy (RMAT) designation from the FDA.