FDA greenlights Ionis' Zanvastro as first therapy for Alexander disease
The U.S. Food and Drug Administration approved Ionis Pharmaceuticals' drug Zanvastro, marking the inaugural disease-modifying treatment for the rare, fatal neurological disorder Alexander disease.
Ionis Pharmaceuticals received FDA clearance for Zanvastro, the first medication designed to alter the course of Alexander disease in both children and adults. A pivotal trial showed that patients receiving the drug maintained their walking speed, while those on placebo experienced a 33% decline. Early-age treatment also hinted at potential improvements in motor function. Safety data indicated fewer serious adverse events among the treated group compared with controls.
Why it matters
It provides the first approved treatment for a deadly, previously untreatable neurological disease.
In this story
