UniQure stock tumbles on FDA comments about gene therapy trial

Investing.com -- UniQure N.V. (NASDAQ: QURE) shares fell 22% Thursday after FDA Commissioner Marty Makary made comments about a rare disease drug trial that appeared to reference the company’s Huntington’s disease gene therapy.

During a CNBC interview with Becky Quick, Makary discussed rare-disease drug approvals and mentioned a product where researchers drilled a hole in patients’ skulls to inject therapy into the brain ventricle. He said the randomized control trial found no benefit at the end of the study period.

"I have a lot of sympathy for those patients. There’s nothing out there to offer them, but we’re not going to go ahead and approve something like that that has morbidity associated with it," Makary said.

STAT Biotech News’ Adam Feuerstein commented on the decline in a post on X, writing UniQure is "down this morning on comments made by FDA Commissioner Marty Makary to CNBC’s Becky Quick."

The FDA has faced criticism over recent rare disease treatment rejections and delays, with multiple biotech companies saying they received different feedback from the agency under the Trump administration compared to the previous one. Makary defended Vinay Prasad, the FDA’s medical and scientific chief, calling him a "genius" facing a "smear campaign."

Prasad oversees gene therapy approvals and has been at the center of controversy over the agency’s approval process. He briefly left the FDA in July amid a dispute over Sarepta Therapeutics Inc.’s gene therapy before returning in August with an expanded role.

Makary said the FDA approval process is straightforward and has gotten faster under a new program to speed approvals of drugs aligned with national strategic interests.

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