The Food and Drug Administration approved Zanvastro, the first FDA-approved treatment for Alexander disease and the first designed to target the abnormal protein buildup that drives the disorder.
Alexander disease is caused by mutations affecting glial fibrillary acidic protein, or GFAP, and can lead to seizures, motor problems, developmental regression and other neurological complications. The drug is administered into the spinal canal every three months.
FDA reviewed clinical data across pediatric and adult patients and granted the therapy several expedited-development designations because of the rarity and seriousness of the disease.