SEP 3 2026
Ionis Pharmaceuticals — Zanvastro (zilganersen) $IONS
for Alexander disease (GFAP-related leukodystrophy)
About
Alexander disease is a rare neurological disorder that damages the protective myelin sheath around the brain's nerve fibers, causing developmental delays, seizures, and loss of motor control. Zanvastro (zilganersen) is an intrathecally delivered antisense oligonucleotide designed to reduce the production of the GFAP protein by targeting its mRNA. By lowering the levels of this disease-driving protein, the drug may help stabilize or improve gross motor function in affected individuals.
Pivotal trial
The purpose of this study is to evaluate the safety and efficacy of zilganersen (ION373) in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD.
Source: ClinicalTrials.gov
Drug Watch — recent news on Zanvastro
- FDA Approves Zanvastro as First Treatment for Alexander Disease - Consultant360
- FDA approves first treatment for Alexander disease, Zanvastro - findarticles.com
- Ionis Has Its First Neurology Launch, But Can Zanvastro Justify Its $285,000 Price? - Yahoo Finance
- Ionis wins FDA approval for Zilganersen to treat Alexander disease - Indian Pharma Post
- FDA Approves Zanvastro for Alexander Disease in Pediatrics & Adults - Pharmaceutical Executive