uniQure Submits Huntington's Disease Gene Therapy for US Approval Following FDA Reversal

Dutch gene therapy developer uniQure has applied for U.S. Food and Drug Administration (FDA) approval of its experimental treatment for Huntington's disease, ifezuntirgene inilparvovec (AMT-130). This submission follows the FDA's reversal of its earlier stance, now accepting existing three-year trial data which indicated the therapy slowed disease progression compared to external databases of Huntington's patients.