STAT+: Ionis wins FDA approval for first drug for Alexander disease, a rare and deadly neurological disorder

The FDA approved Ionis Pharmaceuticals' Zanvastro for children and adults with Alexander disease, a rare and deadly neurological disorder. This is the first disease-modifying therapy for the condition.

In the pivotal trial, walking speed—a common measure of motor function in neurologic disease—remained stable in treated patients, while the control group saw a 33% decline. The study also hinted that treating young children could improve motor function, not just stabilize it. The drug was generally safe, with serious adverse events more common in the control group than among treated patients.