FDA Approves Zanvastro, First Disease-Modifying Treatment for Alexander Disease

The FDA has approved Ionis Pharmaceuticals' Zanvastro (zilganersen), the first and only disease-modifying treatment for Alexander disease. This ultra-rare, progressive leukodystrophy affects both pediatric and adult patients. The RNA-targeted antisense therapy, administered quarterly, demonstrated stabilization of gait speed in older patients and motor improvement in younger children, offering a significant breakthrough for this severe condition.