The FDA Accepts For Priority Review Ionis Pharmaceuticals' New Drug Application For Zilganersen For Alexander disease, With PDUFA Date Of September 22, 2026

The FDA has accepted Ionis Pharmaceuticals' New Drug Application for zilganersen, aimed at treating Alexander disease, and has granted it Priority Review status, with a PDUFA date set for September 22, 2026. The application is based on positive results from a pivotal study showing a 33.3% improvement in gait speed at week 61 compared to control, along with favorable safety and tolerability. Zilganersen has previously received Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations from the FDA.