uniQure Shares Plunge After FDA Setback for Huntington's Drug
Regulator deems current data for gene therapy AMT-130 insufficient for a license application, prompting a wave of analyst price target cuts and delaying a key pipeline asset.
Shares of uniQure N.V. (NASDAQ: QURE) plunged in early trading Thursday after the company disclosed a significant regulatory setback for its flagship gene therapy candidate, AMT-130, for the treatment of Huntington's disease. The U.S. Food and Drug Administration has deemed the company's current data package insufficient to support a Biologics License Application (BLA) at this time, casting uncertainty over the approval timeline for one of the industry's most-watched experimental therapies.
The Amsterdam-based company saw its market value shrink by over $600 million as its stock fell by as much as 40%, reflecting investor concern over the future of a drug central to uniQure’s valuation. The news confirms recent market anxieties that have led to a series of preemptive price target reductions from Wall Street analysts.
In a press release issued before the market opened, uniQure announced that the FDA communicated its position following a pre-BLA meeting on October 29, 2025. The agency stated that the data from the ongoing Phase I/II clinical studies are "unlikely to provide the primary evidence needed to support a BLA submission."
This feedback is a major hurdle for AMT-130, a one-time administered gene therapy designed to combat the neurodegenerative effects of Huntington's disease, a fatal genetic disorder with no cure. For uniQure, the therapy represents the next major pillar of its pipeline beyond its approved hemophilia B treatment, Hemgenix.
"While we are disappointed with the FDA’s current assessment, we remain committed to working collaboratively with the agency to address their feedback," said Matt Kapusta, CEO of uniQure. The company plans to urgently request a follow-up meeting with the FDA in the first quarter of 2026 to discuss a viable path forward.
The regulatory pushback, while formally announced today, has been anticipated by some in the market. In recent weeks, several investment banks have tempered their expectations. Analysts at H.C. Wainwright cut their price target on uniQure from $110 to $70, while RBC Capital adjusted its target to $45 from a previous $65. Stifel and Leerink Partners also made similar downward revisions, all citing the growing regulatory uncertainty for AMT-130.
Despite the lowered targets, many analysts maintain a 'Buy' or 'Outperform' rating, suggesting they believe the setback is a question of timing rather than a fatal flaw in the therapy's clinical profile. The core issue now is what additional data the FDA will require, which could involve longer-term follow-up from existing trial patients or potentially a new, more extensive Phase III study—a costly and time-consuming endeavor.
Huntington's disease is an inherited condition that causes the progressive breakdown of nerve cells in the brain, leading to severe physical and mental deterioration. The potential for AMT-130 to offer a meaningful treatment has made it a cornerstone of uniQure’s research and development efforts. The therapy is designed to deliver a microRNA sequence that silences the huntingtin gene, tackling the root cause of the disease.
For investors, the FDA's stance shifts the focus to uniQure's ability to navigate the complex regulatory process and the financial implications of a prolonged development timeline. The company's cash runway and its revenue from Hemgenix will be under intense scrutiny as it prepares for further dialogue with regulators and potentially more clinical work.