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The U.S. Food and Drug Administration (FDA) provided feedback to uniQure N.V. (NASDAQ:QURE) regarding its investigational gene therapy for Huntington’s disease, causing the stock to decline on Monday.
The company received FDA feedback during a recent pre-Biologics License Application (BLA) meeting regarding AMT-130, an investigational gene therapy for Huntington’s disease (HD).
uniQure believes that the FDA currently disagrees that the data from the Phase 1/2 studies of AMT-130, in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence in support of a BLA submission.
Also Read: uniQure Stock Doubles On Promising Data From Huntington’s Disease Trial
The update marks a key shift from prior communications with the FDA in multiple Type B meetings over the past year. Consequently, the timing of the BLA submission for AMT-130 is now unclear.
uniQure expects to receive final minutes within 30 days of the meeting and plans to urgently interact with the FDA to find a path forward for the timely accelerated approval of AMT-130.
“We are surprised by the FDA’s feedback at the recent pre-BLA meeting, which is a drastic change from the guidance the FDA provided in November 2024 that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis for a BLA submission under the Accelerated Approval pathway,” said Matt Kapusta, CEO at uniQure.
“This news is unexpected, and we are truly disappointed..We strongly believe that AMT-130 has the potential to bring substantial benefit to patients…,” Kapusta added.
In addition to continuing to partner with the FDA on progressing AMT-130 for the treatment of Huntington’s disease, uniQure plans in parallel to progress discussions with other regulatory agencies, including in the European Union and the United Kingdom.
QURE Price Action: uniQure shares were down 53.98% at $31.15 at the time of publication on Monday, according to Benzinga Pro data.
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Posted In: QURE
