STAT+: UniQure plans to seek approval for Huntington’s therapy still blocked by FDA

STAT News · Mar 2, 2026 · Collected from RSS

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Company says agency insists completed trial is insufficient to seek clearance Sipa via AP Images By Adam FeuersteinMarch 2, 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 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. UniQure said Monday that plans to seek approval for its Huntington’s disease treatment with the Food and Drug Administration remain blocked, another setback for a company whose therapy once appeared on a path to approval. Following a meeting with the FDA at the end of January, UniQure said regulators are still not persuaded that data from a completed single-arm clinical trial of its gene therapy, called AMT-130, are sufficient to support a marketing application. “The FDA strongly recommended uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study,” the company said in a statement. 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 View All Plans To read the rest of this story subscribe to STAT+. Subscribe 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.