Ionis Pharmaceuticals announced that 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. ZANVASTRO™ is the first and only disease-modifying treatment for this ultra-rare, progressive, and often fatal neurological disorder.

Key Details

  • Drug Approval: ZANVASTRO™ (zilganersen) is an RNA-targeted medicine designed to reduce the production of glial fibrillary acidic protein (GFAP), the underlying cause of AxD.
  • Clinical Data: The approval was based on a pivotal study that met its primary endpoint, demonstrating a statistically significant stabilization of gait speed compared to control at Week 61 (p=0.041).
  • Commercialization & Regulatory: The drug will be available in the U.S. in the coming weeks. The FDA granted Ionis a Priority Review Voucher. Ionis has a licensing agreement with Recordati for development and commercialization outside the U.S., with regulatory submissions in Europe and Japan expected in 2027.