FDA approves first treatment for rare Alexander disease
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The Food and Drug Administration approved Zanvastro, the first drug to treat Alexander disease, a rare and often fatal neurological disorder. For decades, doctors could only manage the symptoms of this condition, which impacts motor skills, thinking, and bodily functions like breathing.
Researchers at the University of Wisconsin. Madison and Ionis Pharmaceuticals collaborated to create Zanvastro, which targets the protein driving the disease.
The team, led by Albee Messing, discovered the genetic cause of Alexander disease in the late 1990s and then developed a mouse model that mimicked the disease’s effects. This allowed them to test potential therapies, eventually leading to the creation of antisense oligonucleotides, small DNA pieces that reduce production of the problematic GFAP protein. Clinical trials involving 54 patients across 13 sites worldwide demonstrated that Zanvastro stabilized and, in some cases, improved motor function.
The drug is administered via spinal injection every three months. Previously, diagnosing Alexander disease required invasive brain biopsies, but the discovery of the genetic root cause now allows for diagnosis through simple blood tests. Zanvastro is approved for patients of all ages and will be available in the United States soon, with plans for distribution in other countries through an agreement with Recordati, an Italian pharmaceutical company.
