uniQure Shares Tumble 11% Amid FDA Doubts as Volume Slumps to 492nd in U.S. Rankings

Market Snapshot

On November 4, 2025, uniQure (QURE) experienced a sharp decline in both trading volume and stock price, reflecting investor uncertainty following regulatory developments. The company’s shares fell 11.23% for the day, while trading volume dropped 71.43% to $260 million, ranking it 492nd in volume among U.S. equities. This marked a reversal from the stock’s recent performance, which had surged 283% year-to-date and over 1,100% in the past 12 months. The decline was driven by the announcement of unexpected feedback from the U.S. Food and Drug Administration (FDA) regarding its investigational gene therapy, AMT-130, for Huntington’s disease. The FDA’s revised stance on the adequacy of Phase I/II trial data for regulatory submission created immediate market skepticism, triggering the sharp sell-off.

Key Drivers

The FDA’s feedback during a pre-Biologics License Application (BLA) meeting emerged as the primary catalyst for uniQure’s stock collapse. The agency reportedly concluded that data from Phase I/II studies of AMT-130, which had previously been considered sufficient for BLA submission, no longer met regulatory standards. This marked a significant departure from earlier guidance in November 2024, where the FDA had indicated that the data could support a BLA. The shift introduced uncertainty around the timeline for regulatory approval, with the company now anticipating urgent discussions to clarify next steps. The FDA’s feedback also highlighted concerns over the methodology used to compare AMT-130’s efficacy against an external control group, a critical component of the therapy’s regulatory strategy.

The announcement underscored the high-stakes nature of AMT-130’s development, which had been positioned as a potential breakthrough for Huntington’s disease patients. The therapy had previously secured Breakthrough Therapy and Regenerative Medicines Advanced Therapy designations, signaling expedited approval pathways. However, the FDA’s revised stance effectively suspended those expectations, leaving investors with a void in clarity. CEO Matt Kapusta expressed surprise and disappointment, emphasizing the deviation from prior guidance and the need to re-engage with the FDA. The company’s commitment to exploring alternative regulatory pathways was reiterated, but the lack of a concrete timeline for BLA submission left the market exposed to volatility.

The stock’s dramatic 67% premarket drop on November 3, followed by a further 11.23% decline on November 4, reflected the market’s immediate reaction to the regulatory uncertainty. The sell-off erased significant gains accumulated over the past year, which had been fueled by optimism around AMT-130’s potential. Analysts noted that the company’s financial health, already strained by negative margins and declining revenue, added to the vulnerability of its stock. With a market capitalization of $4.17 billion, uniQure’s valuation now faces downward pressure as the likelihood of delayed regulatory milestones becomes more apparent. The stock’s liquidity, evidenced by its 3.2 million average daily trading volume, further amplified the magnitude of the decline.

Beyond the FDA’s feedback, the broader regulatory landscape for gene therapies and the competitive dynamics in the Huntington’s disease space contributed to the stock’s downward trajectory. The FDA’s decision to require additional data for AMT-130 aligns with its cautious approach to novel therapies, particularly those with complex statistical methodologies. Meanwhile, regulatory discussions with the European Union and the United Kingdom, though ongoing, lack the clarity provided by the FDA’s feedback. The company’s pipeline beyond AMT-130, including therapies for refractory temporal lobe epilepsy and Fabry disease, remains underdeveloped relative to its high-profile Huntington’s disease focus, limiting its ability to offset short-term setbacks. The combination of these factors has left uniQure in a precarious position, where investor sentiment hinges entirely on the resolution of its regulatory challenges.

Looking ahead, the company’s next steps will center on clarifying the FDA’s requirements and determining whether additional trials or data analyses are necessary. The anticipated release of final meeting minutes within 30 days could provide critical insights, but until then, the market remains in a state of flux. Legal investigations into potential securities law violations, initiated by multiple law firms, add another layer of uncertainty, though their direct impact on the stock’s performance remains to be seen. For now, the focus remains on the FDA’s evolving stance and the potential for a revised regulatory pathway for AMT-130—a development that could either stabilize or further destabilize uniQure’s stock.