FDA Approves ZANVASTRO™ (zilganersen) as First Disease-Modifying Treatment for Alexander Disease in Pediatric and Adult Patients

The U.S. Food and Drug Administration (FDA) has approved ZANVASTRO™ (zilganersen) for the treatment of Alexander disease (AxD) in both pediatric and adult patients. This marks the first and only disease-modifying treatment for AxD, an ultra-rare, progressive, and often fatal neurological disorder. The approval is based on positive results from a pivotal study.