uniQure said the FDA does not agree that Phase I/II data are sufficient to serve as primary evidence of effectiveness for a marketing application for its Huntington’s disease gene therapy, AMT-130.
uniQure NV (QURE) shares plunged by nearly 45% in Monday’s pre-market trade after the company issued an update about its investigational gene therapy for Huntington’s disease (HD).
uniQure said the FDA does not agree that Phase I/II data, compared with an external control, are sufficient to serve as primary evidence of effectiveness for a marketing application for its Huntington’s disease gene therapy, AMT-130.
“While we did not reach alignment on a submission pathway based on the Phase I/II data, we believe the totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting,” said uniQure CEO Matt Kapusta.
Retail sentiment on Stocktwits around uniQure trended in the ‘extremely bullish’ territory, with message volumes at ‘extremely high’ levels.
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