FDA approves first drug for deadly Alexander disease
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The Food and Drug Administration approved Zanvastro, the first drug to directly treat Alexander disease, a rare and often fatal neurological disorder affecting fewer than one in a million people worldwide.
For three decades, scientists at the University of Wisconsin-Madison and collaborators at Ionis Pharmaceuticals researched the disease, beginning with studies on mice that unexpectedly developed symptoms mirroring the human condition.
Their work identified a mutated gene causing excessive production of a protein called glial fibrillary acidic protein (GFAP) in brain cells. Researchers then developed Zanvastro, which uses antisense oligonucleotides to reduce GFAP production and prevent damaging protein buildup. Clinical trials involving 54 patients at 13 sites showed that Zanvastro stabilized motor function and improved walking speeds in some individuals.
Previously, doctors could only offer supportive care to manage symptoms of Alexander disease, which can include seizures, loss of developmental skills, and difficulty with movement, breathing, and heart rate. The FDA approved Zanvastro for use in patients of all ages and the drug is expected to become available in the United States in the coming weeks.
