Families facing one of the rarest neurological diseases in medicine now have a treatment for the first time, and a price to negotiate. The FDA approved Zanvastro, known in development as zilganersen, for Alexander disease in children and adults, and Ionis Pharmaceuticals has set the list price at $285,000 per dose. Because the drug is given every three months, that works out to roughly $1.14 million a year before rebates or discounts.
Alexander disease affects about one person in every one to three million worldwide. Ionis estimates roughly 300 patients in the United States, while the National Institutes of Health puts the figure at fewer than 1,000. Until now, care meant managing symptoms while the disease progressed.
