UniQure Stock Plummets 67% on Potential FDA Decision. Here’s Why.

The company believes the Food and Drug Administration may deal a setback to one of its investigational therapies. (Dreamstime)

Key Points

• UniQure shares fall sharply after the FDA indicates Phase I/II study data for gene therapy AMT-130 may be insufficient for a BLA.
• AMT-130, an investigational gene therapy for Huntington’s disease, previously received Breakthrough Therapy designation.
• Coming into Monday, uniQure stock has risen 283% this year.

Shares of uniQure tumbled Monday after the biotech company reported a likely regulatory setback for one of its gene therapies.

The Food and Drug Administration no longer agrees that the data from Phase I/II studies of uniQure’s therapy AMT-130 is adequate to support a Biologics License Application, the company said it believes. AMT-130 is an investigational gene therapy for Huntington’s disease, which the FDA granted a Breakthrough Therapy designation earlier this year.

UniQure stock plummeted 67% in premarket trading Monday. Shares closed last Friday up 283% on the year and more than 1,100% over the last 12 months.

Write to Nate Wolf at nate.wolf@barrons.com