FDA approves Ionis' antisense drug as first targeted therapy for Alexander disease

The FDA has approved Zanvastro, an antisense oligonucleotide drug by Ionis Pharmaceuticals, as the first targeted therapy for Alexander disease, an ultrarare genetic neurodegenerative disorder. The drug stabilises motor function by reducing toxic GFAP protein buildup and is approved for all ages, marking Ionis’ entry into neurology with potential for further approvals in rare neurodegenerative diseases.

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