STAT+: Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval

STAT PlusBiotech Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval Closely watched therapy would be first genetic treatment for condition Manage alerts for this article Email this article Share this article By Adam FeuersteinJune 17, 2026 Adam Feuerstein, a senior writer and biotech columnist, is the author of Adam’s Biotech Scorecard, a subscriber-only newsletter about the crossroads of drug development, business, Wall Street, and biotechnology. Adam Feuerstein[email protected]Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54. The Food and Drug Administration has reversed its opposition to a closely watched experimental treatment for Huntington’s disease, clearing a path for its maker, the biotech company UniQure, to file for U.S. approval, the company said Wednesday. UniQure will submit a marketing application in the third quarter seeking accelerated approval for the treatment, called AMT-130. The decision comes after a recent meeting with FDA officials during which the agency agreed that a three-year analysis of an early-stage study that showed a benefit for patients with Huntington’s was “acceptable” to support a marketing application, the company said. Advertisement These are the same AMT-130 data that former FDA officials, most notably Commissioner Marty Makary and Vinay Prasad, the agency’s top regulator of cell and gene therapies, previously concluded were insufficient to support a marketing application. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in Monthly $39 Totals $468 per year $39/month Get Started Totals $468 per year Starter $30 for 3 months, then $399/year $30 for 3 months Get Started Then $399/year Annual $399 Save 15% $399/year Get Started Save 15% 11+ Users Custom Savings start at 25%! Request A Quote Request A Quote Savings start at 25%! 2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+. Subscribe Log In biotechnology, chronic disease, drug development, Pharmaceuticals, STAT+ Submit a correction requestReprints Adam Feuerstein Senior Writer, Biotech Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54. STAT+ Newsletter Senior writer Adam Feuerstein's unfiltered, uncompromising analysis on the biotech world Recommended The Readout June 17, 2026 STAT Plus: FDA appears open to Moderna’s flu vaccine ahead of adcomm Biotech June 17, 2026 STAT Plus: A prominent VC explains why she’s against U.S. restrictions on investment in China’s drug industry Advertisement The Readout June 16, 2026 STAT Plus: ARCH-launched Neumora stops depression program Biotech June 16, 2026 STAT Plus: Verge Labs’ new AI model solves patient stratification problems for neurology clinical trials Politics June 15, 2026 STAT Plus: Trump’s obesity drug plan creates a temporary Medicare program that may be hard to end Subscriber Picks