NEW YORK, March 4 (Reuters) – UniQure’s trial design for its gene therapy for Huntington’s disease did not offer sufficient proof that the drug slowed disease progression, a top U.S. Food and Drug Administration official said on Wednesday, while defending the agency’s call for a new placebo-controlled study.
The official, who spoke on the condition of anonymity, said the FDA has historically rejected studies that are not randomized and placebo-controlled when considering how to approve potential Huntington’s disease treatments.
On Monday, UniQure, a Dutch drugmaker, said the drug regulator called for a new study to support the approval of its gene therapy for the disorder based on a placebo, a study analysts said would be difficult to conduct. Huntington’s is a rare, inherited neurodegenerative disorder.
The company’s U.S.-listed shares closed on Wednesday at $9.04, down sharply from about $15 before the news.
UniQure declined to provide an immediate comment.
UniQure’s studies last year showed a statistically significant improvement in slowing the progression of the disease after three years based on a rating scale when compared with patients from a dataset of external Huntington’s patients.
The FDA official said the comparison was not acceptable.
In late 2024, UniQure said it reached an agreement on its key application components for an accelerated approval review by the FDA of the therapy.
The agency never told UniQure that the study would be enough for approval but gave its typical advice, the FDA official said.
“You can run whatever study you want,” the official said. “We will take a look at such data with a very critical eye, and we will evaluate it in the context.”
During a conference call to discuss financial results, UniQure medical chief Walid Abi-Saab described the FDA as having requested that it “superficially drill a hole on the skull,” a description the FDA official disputed.
On Wednesday, Department of Health and Human Services spokesman Andrew Nixon called the UniQure statement false on social media.
The FDA official said the agency told the company that a randomized placebo-controlled study could be based on a sham procedure in which the patient would receive a small scalp incision.
The gene therapy is administered after neurosurgery, which involves drilling through the skull, directly into the brain’s striatum.
(Reporting by Caroline Humer; Editing by Thomas Derpinghaus)
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