UniQure files FDA priority review request for Huntington’s gene therapy AMT-130

UniQure, a Dutch biotech, announced on Wednesday that it has submitted applications to the U.S. Food and Drug Administration seeking a priority review for its gene therapy AMT-130, which targets the underlying cause of Huntington’s disease.

The company also filed a parallel submission with regulators in the United Kingdom. The filings rely on three-year data that the firm previously disclosed, showing that AMT-130 can markedly slow disease progression in patients.

UniQure is requesting accelerated approval, meaning the FDA could grant conditional marketing authorization now but would require additional confirmatory studies to verify clinical benefit later. The move follows a turbulent year of interactions with U.S. regulators and a prior surge in the company’s share price after earlier positive trial signals.