ZANVASTRO™ (zilganersen) approved by the FDA as the first and only disease modifying treatment for Alexander disease (AxD) in pediatric and adult patients
Ionis announced that the FDA approved ZANVASTRO (zilganersen) as the first and only disease-modifying treatment for Alexander disease (AxD) in pediatric and adult patients. AxD is an ultra-rare, progressive, often fatal neurological disorder affecting motor, cognitive, autonomic, and gastrointestinal function.
Why it matters — This FDA approval gives Ionis its first and only disease-modifying treatment for Alexander disease, marking a significant regulatory milestone for the company's pipeline.