Ionis Pharmaceuticals announced additional positive results from the pivotal study of zilganersen in Alexander disease, meeting its primary endpoint and showing favorable safety, with a PDUFA action date of September 22, 2026.
Key takeaways
Zilganersen met its primary endpoint in patients ≥5 years, showing statistically significant stabilization of gait speed at Week 61.
The study demonstrated favorable safety and tolerability, with serious adverse events occurring less frequently in the zilganersen group.
Key secondary endpoints across patient and clinician assessments consistently favored zilganersen over control.
Zilganersen is under FDA Priority Review with a PDUFA action date of September 22, 2026.