TLDR
- QURE stock crashed 67% in premarket trading on November 3, 2025
- FDA reversed its position on AMT-130 Phase I/II data adequacy for licensing
- AMT-130 treats Huntington’s disease and holds Breakthrough Therapy status
- Stock had surged 283% year-to-date before the regulatory setback
- Company pledges to work with FDA on alternative approval pathway
UniQure stock experienced a dramatic selloff on November 3, 2025, following unexpected regulatory news. The biotech company revealed the Food and Drug Administration changed its stance on critical data for its Huntington’s disease treatment.
Shares fell 67% in premarket trading. The decline erased much of the stock’s impressive gains from earlier in 2025.
FDA Changes Course on AMT-130 Data
The FDA no longer considers Phase I/II study data sufficient for a Biologics License Application. The agency previously indicated this data could support the filing.
UniQure’s AMT-130 is a gene therapy candidate for Huntington’s disease. The treatment received Breakthrough Therapy designation from the FDA in 2025.
The company disclosed the regulatory update after a pre-BLA meeting with federal regulators. UniQure had planned to use Phase I/II data compared against external controls for its application.
The FDA’s reversal creates uncertainty about the BLA submission timeline. UniQure has not announced when it might file the application.
Stock Performance Before the Drop
QURE stock had been one of 2025’s top performers. Shares closed Friday up 283% for the year.
The 12-month returns were even stronger. UniQure stock gained over 1,100% in the trailing year.
The stock’s rally reflected investor confidence in AMT-130’s approval prospects. Monday’s news significantly dampened that optimism.
What Happens Next for UniQure
The company says it will collaborate with the FDA to identify an approval pathway. UniQure is also engaging with other international regulatory agencies.
Management emphasized AMT-130’s potential to help Huntington’s disease patients. The company appears determined to advance the therapy despite the setback.
UniQure operates a broader pipeline beyond AMT-130. The biotech is developing treatments for temporal lobe epilepsy, ALS, and Fabry disease.
The company previously earned approval for a hemophilia B gene therapy. UniQure focuses on gene therapy treatments for serious medical conditions.
Analysts rate QURE stock as a Hold with a $67 price target. The company’s market capitalization is $4.17 billion.
Average daily trading volume reaches 3.2 million shares. Technical indicators showed a Buy signal before Monday’s drop.
UniQure faces financial headwinds beyond the regulatory news. The company reports declining revenues and operates with negative profit margins.
The biotech announced the FDA’s position change on November 3, 2025, following its pre-BLA regulatory meeting.
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