After 30 years of research, first drug for the rare Alexander disease

People with Alexander disease now have, for the first time, a medicine designed to act on the disease process itself. The U.S. Food and Drug Administration approved Zanvastro (zilganersen) from Ionis for children and adults, trade media reported in early September. On Oct. 12, the University of Wisconsin–Madison described how roughly 30 years of its research paved the way. “This is a wonderful and long-awaited day for the Alexander disease community,” said Albee Messing, professor emeritus and former director of the Waisman Center, whose lab laid the groundwork.

Alexander disease is extremely rare, affecting about one in 1 million to 3 million people, according to American Pharmaceutical Review. It is caused by changes in the GFAP gene that lead a protein to build up in certain brain cells called astrocytes. The disease can begin in infancy, childhood or adulthood and can affect movement, thinking and digestion. Zanvastro is designed to reduce production of that protein. It is injected into the spinal canal once every three months.

The approval is based on a study of 54 participants aged 1.5 to 53 at 13 sites in eight countries. In patients aged five and older, walking speed stabilized at week 61 compared with a control group. Common side effects included vomiting, back pain and headache; the label warns of aseptic meningitis. Recordati holds the rights outside the United States and expects filings in Europe and Japan in 2027.