FDA Grants uniQure Priority Review for First Huntington's Gene Therapy While Inovio Wins Accelerated Approval for Rare Airway Disease

The FDA accepted uniQure's Biologics License Application for ifezuntirgene inilparvovec, an investigational gene therapy for Huntington's disease, under the Accelerated Approval pathway and granted Priority Review. The agency set a PDUFA target action date of May 1, 2027. If approved, ifezuntirgene inilparvovec would become the world's first disease-modifying treatment for Huntington's disease, a fatal inherited neurological disorder with no currently approved therapies that alter its course.

The therapy, also known as AMT-130, already holds three FDA designations: Breakthrough Therapy, Regenerative Medicine Advanced Therapy, and Fast Track, making it the first Huntington's candidate to have received both Breakthrough Therapy and RMAT designations. The BLA is supported by data from uniQure's Phase I/II clinical studies. Shares of uniQure rose approximately 5.7% in premarket trading following the announcement.

Separately, Inovio Pharmaceuticals received FDA accelerated approval for VOCERYQ, a DNA immunotherapy to reduce the frequency of surgery in adults with recurrent respiratory papillomatosis, a rare condition caused by HPV that leads to recurring growths in the airway. VOCERYQ, previously known as INO-3107, is described as the first FDA-approved DNA medicine, validating Inovio's platform technology. The approval drove Inovio shares up roughly 260% in premarket trading on November 2.