IONS Secures FDA Nod for Rare Brain Disorder Drug Zanvastro

Ionis Pharmaceuticals announced FDA approval of Zanvastro (zilganersen), the first disease-modifying treatment for rare neurological condition Alexander disease (AxD) in both pediatric and adult patients. The approval was supported by Phase III results showing statistically significant stabilization in gait speed. Ionis also received a rare pediatric disease priority review voucher. However, commercial potential is limited due to the ultra-rare nature of the condition affecting approximately 1 in 1-3 million people worldwide.