Zilganersen targets protein buildup that drives Alexander disease
— Zilganersen targets protein buildup that drives Alexander disease
by Judy George, Deputy Managing Editor, MedPage Today
September 4, 2026
• 2 min read
The FDA approved zilganersen (Zanvastro) to treat Alexander disease in pediatric and adult patients, the agency announced Thursday.
Ionis wins FDA clearance for Alexander disease drug, company's first solo neurology approval
Ionis’ rare neurology drug approved; Pfizer inks an ADC deal
Ionis’ ‘breakthrough’ Alexander disease approval establishes pillar of rare disease pipeline
Mimrylo snags FDA approval for first-of-its-kind polycythemia vera treatment
The FDA approves Ionis' Zanvastro as the first treatment for Alexander disease, clearing the way for the company's first solo…
Zanvastro is now the first approved medicine for Alexander disease. Elsewhere, an immune system specialist posted positive…
Ionis announces license agreement with Recordati for zilganersen in Alexander disease (AxD) in all countries outside the U.S.
Galibra Neuroscience Receives FDA Orphan Drug and Rare Pediatric Disease Designations for SSADH Deficiency Gene Therapy
After Jenny Pearson’s daughter Elise first had a seizure at 16 months old, an MRI came back clean—her brain “looked beautiful,”…
FDA grants Ultragenyx's Genglycos accelerated approval, its first gene therapy, for GSDIa in patients 8 and up; list price $2.7M.
