Ionis receives US FDA breakthrough therapy designation for zilganersen for Alexander disease

Ionis Pharmaceuticals

2 December 2025 - On track to submit new drug application in Q1, 2026.

Ionis Pharmaceuticals today announced that the US FDA has granted breakthrough therapy designation to zilganersen for the treatment of Alexander disease, a rare, progressive and often fatal neurological condition.

Read Ionis Pharmaceuticals press release

Michael Wonder

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Michael Wonder