The path to approval for uniQure’s gene therapy candidate for Huntington’s disease is in doubt as the US Food and Drug Administration (FDA) has brought into question the adequacy of company’s Phase I/II trial data, which sent stocks plummeting by almost 66% when the market opened on 3 November.
The single-use gene-therapy candidate was pegged to be the first ever therapy approved for Huntington’s following a positive mid-stage trial result, which saw the Dutch biotech’s stocks soar by 247%, in late September.
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Fresh discussions with the FDA indicated that the Phase I/II data for AMT-130 would not be sufficient for a biologics license application (BLA), in what uniQure called a “drastic change” from previous guidance from several Type B meeting.
“This news is unexpected, and we are truly disappointed for people living with HD, who have no disease-modifying treatment options for this devastating disease,” said uniQure CEO Matt Kapusta.
While uniQure’s stock has slightly risen since then, opening at $30.81 today (4 November), investors appeared spooked by the news.
“We believe that the FDA’s commentary on AMT-130 suggests the agency is behaving more rigidly regarding the regulation of gene/cell therapy programs for rare diseases,” said Sami Corwin from the equity research analyst firm William Blair.
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By GlobalDataIn a note, Corwin added that this could have negative impact for other cell and gene therapy companies that are investigating candidates in single arm trials, such as Neurogene, Cabaletta Bio, Kyverna Therapeutics and Rocket Pharmaceuticals. However, the FDA’s concerns appear to be primarily focused on uniQure having not proposed the statistical analysis plan prior to the start of the trial, added Corwin in the note.
In the Phase I/II study (NCT05243017), high-dose AMT-130 slowed disease progression by 75% at 36 months as measured by the unified Huntington’s disease rating scale (cUHDRS), significantly more than the external control groups. cUHDRS looks at several key aspects of motor and cognitive function to determine the progression of Huntington’s disease. In the wake of these topline findings, uniQure had anticipated launching the disease-modifying therapy (DMT) by the end of 2026 under an accelerated approval pathway, having been granted an FDA breakthrough designation in April 2025.
uniQure will be mulling over next steps as it awaits final minutes from the pre-BLA meeting, expected within 30 days. In parallel, the company plans to progress approval discussion with regulatory agencies in the EU and UK. The timing of a BLA submission is now unclear, according to uniQure.
In a note, Corwin also highlighted an important difference between the most recent pre-BLA meeting and prior positive discussions between uniQure and the FDA regarding AMT-130. Perhaps crucially, Nicole Verdun, previously the director of the cell and gene therapy office, was not in attendance, having been placed on administrative leave back in June 2025. It was also pointed out that Vinay Prasad head of the FDA’s Center for Biologics Evaluation and Research, was also not in the meeting due his abrupt departure in July and subsequent return to the role just weeks after.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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