UniQure Stock Plummets 65% on FDA Reversal for Huntington's Drug
The U.S. regulator unexpectedly deemed Phase I/II trial data for gene therapy AMT-130 insufficient for an accelerated approval submission, creating significant delays.
Shares of uniQure N.V. (NASDAQ: QURE) collapsed in pre-market trading on Monday after the company disclosed a major regulatory setback for its pivotal Huntington's disease gene therapy, AMT-130. The U.S. Food and Drug Administration (FDA) unexpectedly reversed its previous guidance, stating that the current clinical data is insufficient to support an accelerated approval pathway, jeopardizing the treatment's route to market.
The news sent the biotech firm's stock plummeting more than 65% to $23.40 in pre-market trading, wiping out a significant portion of its market capitalization and reflecting deep investor concern over the future of what was considered a flagship asset in uniQure's pipeline.
Huntington's disease is a rare, fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain, for which there are currently no disease-modifying treatments. UniQure's AMT-130, which has received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, was seen as a promising candidate to address this significant unmet medical need.
The setback stems from a recent pre-Biologics License Application (BLA) meeting with the FDA. In a statement, uniQure revealed the agency no longer agrees that data from its ongoing Phase I/II studies, compared against a natural history external control group, could serve as the primary evidence for a BLA submission. This marks a stark departure from guidance the company said it received just a year prior.
"We are surprised by the FDA’s feedback... which is a drastic change from the guidance the FDA provided in November 2024," said Matt Kapusta, chief executive officer of uniQure, in a press release. "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."
The FDA's revised stance means the timeline for a BLA submission is now highly uncertain. For biotech companies like uniQure, the accelerated approval pathway is a critical mechanism to bring promising therapies for serious conditions to patients sooner, based on data that suggests a likely clinical benefit. A delay not only impacts patients but also adds significant costs and uncertainty to the development process.
Analysts were quick to react to the news, with the development casting a cloud over the drug's path to approval. According to Investing.com, the unexpected shift from the regulator has forced a reassessment of the company's short-term prospects.
Despite the setback, uniQure's management affirmed its commitment to the program. "We strongly believe that AMT-130 has the potential to bring substantial benefit to patients, and we remain fully committed to working with the FDA to determine the best path forward," Kapusta added. The company plans to "urgently interact" with the FDA upon receiving the final meeting minutes and will continue to advance regulatory discussions in the European Union and the United Kingdom.
The market's severe reaction underscores the high-stakes nature of biotech drug development, where regulatory decisions can dramatically alter a company's trajectory. Investors will now be closely watching for the formal meeting minutes and any subsequent updates from uniQure's engagement with the FDA to find a viable path forward for AMT-130.