Posted on 8th September 2026

FDA approve first treatment for Alexander Disease in the US

We welcome the news that The U.S. Food and Drug Administration (FDA) has approved ZANVASTRO™ (zilganersen) for people living with Alexander disease in the United States, making it the first approved treatment for this rare leukodystrophy.

You can read the full announcement from Ionis here. Please note, this document contains the complete details, including the important safety information, warnings and precautions.

The Alexander disease Community Statement from Ionis can be found here.

The FDA announcement can be found here.