FDA approves Ionis' Zanvastro as first treatment for rare Alexander disease
The FDA approved Zanvastro (zilganersen) from Ionis as the first disease-modifying therapy for Alexander disease, a rare GFAP-related brain disorder, for adults and children. The intrathecal antisense oligonucleotide lowers abnormal GFAP protein and trials showed stabilization or improvement in motor function with a favorable safety profile, though administration requires specialized settings.
Health · 14 sources · Sep 9, 2026Read itThe journal
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About Ionis Pharmaceuticals
Ionis Pharmaceuticals, Inc. is a biotechnology company that specializes in discovering and developing antisense therapy, as well as RNA interference and CRISPR therapeutics. The company was founded in 1989 is based in Carlsbad, California. The company was previously known as Isis Pharmaceuticals until December 2015.
Everything about Ionis Pharmaceuticals →- Country
- United States
- Headquarters
- Carlsbad
- Inception
- 1989
- Website
- http://www.ionispharma.com/