June 17 (Reuters) – UniQure said on Wednesday the U.S. Food and Drug Administration has agreed existing trial data could support an accelerated-approval filing for its Huntington’s treatment, sending the Dutch drugmaker’s U.S.-listed shares up more than 70% in premarket trading.
The decision is a significant reversal by the agency. FDA officials had indicated late last year that available data was insufficient to support a filing, with one senior regulator describing uniQure’s gene therapy, AMT-130, as a “failed product.”
Data from an early-to-mid-stage trial published last year showed patients who received a high dose of AMT-130 saw a 75% reduction in disease progression in a three-year analysis, based on a widely used clinical scale.
The FDA has now told the company following a recent formal meeting that the three-year analysis would be sufficient as the main basis for a marketing application, uniQure said.
The agency has also agreed to work with uniQure to finalize the design of a required confirmatory study before the filing, including using patients on standard treatment as a comparison instead of a sham procedure.
UniQure plans to submit its application in the third quarter of 2026.
“We think this is great news for QURE and may suggest that the pendulum between regulatory leniency versus inflexible scientific rigor is swinging back to the former, now that Vinay Prasad and Marty Makary have left the FDA,” RBC Capital Markets analysts said in a note.
The agency appears to be trying to repair relations with the rare-diseases sector, with acting FDA Commissioner Kyle Diamantas earlier this month meeting with rare disease advocacy groups in what one attendee called a “breath of fresh air.”
The accelerated approval pathway is designed to speed up access to treatments for serious conditions with no good alternatives.
Huntington’s disease is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain, leading to movement, cognitive and psychiatric problems. It currently has no approved disease-modifying therapy.
(Reporting by Kamal Choudhury in Bengaluru; Editing by Jonathan Ananda)
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