FDA Approves First Drug to Treat Rare Neurological Disorder, Alexander Disease

The U.S. Food and Drug Administration (FDA) has approved Zanvastro (zilganersen) injection, marking the first-ever approved treatment for Alexander disease in both pediatric and adult patients. This antisense oligonucleotide therapy directly targets the underlying cause of the rare, progressive neurological disorder by reducing the production of the abnormal glial fibrillary acidic protein (GFAP) that accumulates in the brain. Clinical trials showed Zanvastro improved walking speed in patients aged 5 and older and motor skills in children aged 2 to 4.