Sep 4, 2026 —
Ionis Pharmaceuticals has received U.S. Food and Drug Administration (FDA) approval for zilganersen, to be marketed as Zanvastro, for Alexander disease, a rare neurodegenerative disorder — the first disease-modifying therapy approved in this indication.
Zanvastro is an antisense oligonucleotide designed to reduce production of glial fibrillary acidic protein (GFAP), whose abnormal accumulation in astrocytes drives Alexander disease. The approval covers both pediatric and adult patients.
The approval adds to the growing set of RNA-targeting genetic medicines reaching patients with rare neurological diseases that previously had no disease-modifying options.
