FDA reverses course, clears UniQure to file Huntington's gene therapy
UniQure said on June 17 that the FDA has reversed its earlier opposition and agreed that data on its experimental gene therapy AMT-130 are “acceptable” to support a marketing application, STAT reported . The company plans to submit for accelerated approval in the third quarter of 2026. If cleared, AMT-130 would be the first therapy to treat the underlying course of Huntington’s disease, a fatal inherited neurodegenerative disorder.
The decision follows a recent meeting between the company and the agency. Former FDA Commissioner Marty Makary and Vinay Prasad, the agency’s top cell and gene therapy regulator, had previously concluded the same three-year data from an early-stage study were insufficient. The shift turns on the agency’s willingness to treat a small, uncontrolled trial as adequate for a disease with no existing disease-modifying option — a judgment that will draw scrutiny given the regulators’ earlier skepticism and the broader debate over the evidence bar for accelerated approvals.