UniQure's Huntington's gene therapy reaches the FDA, three months after a reversal

uniQure said Wednesday it has asked the FDA to review ifezuntirgene inilparvovec (AMT-130), an experimental gene therapy for Huntington's disease, a fatal inherited brain disorder with no approved treatment that slows or stops it. The agency had told the company earlier this year that its early-stage data were not enough to support a filing. In June, it reversed course and let the BLA in.

A biologics license application, or BLA, asks the FDA to consider a therapy for marketing approval. uniQure is seeking accelerated approval, a pathway that lets the agency act on a surrogate endpoint reasonably likely to predict clinical benefit, with confirmatory evidence required afterward. For a fatal neurodegenerative disease with no disease-modifying alternative, the FDA's own type C meeting minutes still flag unresolved scientific questions about the dataset.

uniQure also filed in parallel with the U.K. MHRA and raised $259M earlier this year, material context for a gene therapy whose launch economics, from manufacturing to single-dose pricing, are capital-intensive.

The next decision points are datable: FDA acceptance of the filing, an advisory committee if the agency convenes one, and a target decision date under the user-fee clock. Acceptance puts a calendar on the review; a refuse-to-file letter resets the clock and reopens the question the June reversal tried to close.