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On September 3, 2026, the U.S. FDA approved ZANVASTRO (zilganersen) for the treatment of Alexander disease in…
IONSIonis Pharmaceuticals Inc
On September 3, 2026, the U.S. FDA approved ZANVASTRO (zilganersen) for the treatment of Alexander disease in pediatric and adult patients. This marks the first and only disease-modifying treatment for this ultra-rare neurological disorder. The approval is based on pivotal study results demonstrating statistically significant stabilization of gait speed compared to control at Week 61. Ionis received a Priority Review Voucher with the approval. ZANVASTRO will be available in the U.S. in the coming weeks, while Recordati holds exclusive rights for commercialization outside the U.S. under a June 2026 license agreement. Ionis Pharmaceuticals is a biotechnology company focused on developing RNA-targeted medicines.