FDA nod for rare neurology drug
The FDA has approved Ionis Pharmaceuticals' Zanvastro (zilganersen), the first disease-modifying drug for Alexander disease, a rare progressive and often fatal neurological condition affecting about one person per 1-3 million. The antisense oligonucleotide targets RNA encoding glial fibrillary acidic protein (GFAP), reducing its overproduction in astrocytes, where excess GFAP forms clumps that disrupt myelination. Patients suffer impaired motor function, seizures, intellectual disability and developmental delays.
Why it matters: First disease-modifying therapy approved for a rare, previously untreatable neurological condition.