FDA approves Zanvastro, first therapy targeting protein buildup in Alexander disease

The U.S. Food and Drug Administration has approved Zanvastro (zilganersen) injection for the treatment of Alexander disease in both pediatric and adult patients.

Zanvastro is the first FDA-approved therapy for the condition and the first to directly target the abnormal protein buildup that drives the disease.

Alexander disease is a rare, progressive neurological disorder caused by mutations that produce abnormal glial fibrillary acidic protein, leading to seizures, loss of milestones, walking difficulty, muscle weakness, and increased brain pressure.

The drug is an antisense oligonucleotide designed to reduce production of the faulty GFAP protein before it accumulates and causes further damage.