UniQure shares fall after FDA comments raise questions over AMT-130 review path

UniQure's stock fell ~28% on Thursday after FDA Commissioner Marty Makary appeared to reference the company's Huntington's disease therapy, AMT-130, and since the news broke, the stock has lost more than 65% of its value from recent highs. His remarks, defending the agency's recent rejections of rare disease drugs, included a description of a procedure involving a "burr hole" drilled into the skull to deliver a therapy, raising questions about AMT-130's invasive delivery method.

In April, the company secured a special classification for AMT-130 that promised a faster review. In September, the company unveiled mid-stage trial results that showed a 75% slowing of disease progression, and the plan was to file a Biologics License Application in 2026. A Type A meeting with the FDA was scheduled for early January to discuss that submission.

The pivotal study met its primary endpoint, showing high-dose AMT-130 slowed Huntington's disease progression by 75% at 36 months compared to controls. The effect was confirmed on a key secondary measure and supported by favorable biomarker trends. The data were from a Phase I/II trial.

In recent months, the agency has been promoting a framework allowing approval based on a plausible mechanism for ultra-rare diseases where traditional trials are infeasible. The approach requires substantial evidence of a therapy's biological plausibility and target engagement.

With a market cap of roughly $1.44 billion, the shares trade at a steep discount to their recent highs. The current sell-off reflects market concern that the FDA's comments could complicate the regulatory path for a therapy that uses brain delivery, even as the company prepares for further dialogue with the agency.