Agency ‘not convinced’ UniQure Huntington’s therapy has benefit

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 concluded that an experimental treatment for Huntington’s disease developed by UniQure was not providing benefit for patients based on existing clinical data, a senior FDA official told STAT, explaining the agency’s decision to block the company from submitting a marketing application. 

Agency reviewers “are not convinced there’s any therapeutic benefit of the product,” the official said, speaking on condition of anonymity. 

“If we felt there was any therapeutic benefit, we, of course, would approve it. But they’re not persuaded,” the official added. 

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