FDA approves Zanvastro, first drug for Alexander disease

The FDA has approved Zanvastro, the first and only disease-modifying treatment for Alexander disease, a rare and often fatal neurological disorder. Developed by Ionis Pharmaceuticals based on three decades of research at the University of Wisconsin-Madison, it is injected into the spinal canal every three months.

• Zanvastro is the first approved disease-modifying treatment for Alexander disease
• It uses antisense oligonucleotides to reduce production of the GFAP protein
• The drug is given as a spinal canal injection once every three months
• Trials showed stabilized motor function and some improvement in movement