FDA approves first-ever drug to target rare neurological disorder Alexander disease
The drug zilganersen targets GFAP directly; until now, treatments for the neurological condition have focused on managing symptoms
The US Food and Drug Administration has approved the first drug to directly treat Alexander disease, a rare genetic neurological disorder that affects fewer than one in a million people worldwide and is often fatal. The drug, zilganersen (brand name Zanvastro), developed by Ionis Pharmaceuticals, targets GFAP directly, reducing the amount the body produces.