Ionis reports positive pivotal trial results for zilganersen in Alexander disease

Filed April 21, 2026 · Period ending April 21, 2026 · ~1 min read

Key Changes

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  Zilganersen met primary endpoint, showing statistically significant stabilization of gait speed (33.3% improvement, p=0.041) in patients 5+ years with Alexander disease, a rare neurological disorder with no approved treatments.

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  FDA Priority Review underway with action date of September 22, 2026, meaning potential approval decision within five months if agency maintains timeline.

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  Younger children (ages 2-4) showed 22.9-point improvement in gross motor function versus control (p=0.034), extending potential benefit beyond primary study population.

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