UniQure Pursues FDA Approval for Huntington’s Gene Therapy

The path to submission marks a sharp pivot from earlier this year. In March, federal regulators openly criticized the company, arguing that its clinical evidence—which relied on comparisons to an external database rather than a placebo-controlled trial—was insufficient. Former FDA leadership previously insisted that a sham surgery was necessary to prove the treatment’s efficacy, a requirement UniQure rejected as unethical given the complexity of the brain surgery involved.

Following the departure of former agency leadership in May, the regulatory environment shifted. UniQure reported that the FDA now views its long-term data as a viable foundation for an application. The news triggered a significant market reaction, with company shares climbing 70% on Wednesday. The therapy, designed to combat the hereditary destruction of nerve cells, demonstrated a 75% reduction in disease progression during initial trials, providing the clinical basis for the company’s upcoming regulatory push.