FDA Approves Zanvastro, First Drug for Alexander Disease

The FDA has approved Zanvastro from Ionis Pharmaceuticals, the first and only treatment that changes the course of Alexander disease, a rare and often fatal neurological disorder. The drug is given as a spinal canal injection every three months and will reach the US in the coming weeks, with international use handled by Recordati.

• Zanvastro is the first treatment targeting the cause of Alexander disease
• In trials of 54 patients, walking speed and motor function improved or stabilized
• Given as a spinal injection every three months, approved for all ages
• US launch in coming weeks; Recordati will handle use abroad