FDA approves Zanvastro, first Alexander disease drug | The Locally Times

Published 9/19/2026, 9:16:04 AM UTC · The Locally Times

The Food and Drug Administration approved Zanvastro, the first disease-modifying treatment for Alexander disease.

The Food and Drug Administration approved Zanvastro, the first drug to treat Alexander disease, a rare, progressive and often fatal neurological disorder that can affect motor, cognitive and autonomic functions like heart rate and breathing. The drug is the first and only disease-modifying treatment for Alexander disease, and until now treatment has been limited to symptom management. Zanvastro directly targets the protein that drives the disease and was developed in collaboration with Ionis Pharmaceuticals from research done at the University of Wisconsin-Madison's School of Veterinary Medicine and Waisman Center. The research originated in the laboratory of Albee Messing, professor emeritus of comparative biosciences and former Waisman Center director, who first discovered the genetic root cause of the disease in the late 1990s with Michael Brenner, professor emeritus of neurobiology at the University of Alabama at Birmingham. In early experiments, Messing and Brenner designed a mouse to overexpress the gene that makes glial fibrillary acidic protein (GFAP), and the mice developed clumps of proteins in brain cells called astrocytes, known as Rosenthal fibers, a classic feature of Alexander disease. Further genetic analysis of individuals with the disease confirmed GFAP genetic mutations as the root cause. During clinical trials, individuals with Alexander disease treated with Zanvastro showed stabilization of motor function, and in some cases, patients' motor function improved. Alexander disease is an ultra-rare disease that affects fewer than one in a million people worldwide, with symptoms that can include seizures, loss of developmental skills, mobility challenges, difficulties with eating and speaking, and increased pressure in the brain. Initial signs of Alexander disease can appear from infancy through adulthood and may vary depending on age of onset.