FDA approves Zanvastro, first drug for rare Alexander disease

Ionis Pharmaceuticals won FDA approval for Zanvastro, the first disease-modifying therapy for Alexander disease, a rare and deadly neurological disorder. In a pivotal trial, treated patients maintained walking speed while the control group saw a 33% decline. The drug is approved for children and adults, and early data hint it may improve motor function in young children, not merely stabilise it.