- Zanvastro is the first FDA-approved treatment for Alexander disease and directly targets the abnormal GFAP protein buildup that drives the disease.
- The approval covers patients from infancy through adulthood and is supported by a randomized study in 49 patients aged 2 and older plus an open-label substudy in four patients under age 2.
- In the pivotal study, treated patients aged 5 and older with baseline walking difficulty had significantly better walking speed at 61 weeks than untreated patients.
Quotes
“Today’s approval is a landmark moment for this community, offering the first therapy that addresses the underlying cause of this rare and serious disease.”