uniQure falls after FDA commissioner’s comments on rare disease therapy

uniQure NV shares dropped sharply on Tuesday after comments from FDA Commissioner Robert Califf raised concerns about the regulatory pathway for gene therapies targeting rare diseases. Califf's remarks, made during a conference, suggested that the agency is grappling with how to evaluate the long-term safety and efficacy of these novel treatments, leading to investor apprehension about the future approval prospects for uniQure's lead candidate, etranacogene dezaparvovec, which is being developed for hemophilia B.