Zanvastro (zilganersen) is an antisense oligonucleotide indicated for the treatment of Alexander disease in pediatric and adult patients. It targets glial fibrillary acidic protein (GFAP) messenger RNA to reduce the production of abnormal GFAP protein, which accumulates in the brain and contributes to disease progression. Administered by intrathecal injection, Zanvastro is designed to target an underlying disease mechanism associated with this rare neurological disorder.
As the first FDA-approved treatment for Alexander disease, Zanvastro represents an important advancement in the treatment of rare neurological diseases. By reducing abnormal GFAP production, it aims to address a key biological driver of the condition and potentially help preserve neurological function. Its approval provides a targeted treatment option for patients across pediatric and adult populations living with this progressive genetic disorder.
