FDA approves Zanvastro, 1st drug directly targeting Alexander disease
Technology
Big news: The FDA has greenlit Zanvastro, the first-ever drug to attack Alexander disease directly, a super-rare neurological disorder that affects fewer than 1 in 1 million people.
Made by Ionis Pharmaceuticals, this drug targets the root cause by lowering levels of a protein (GFAP) that builds up and damages the brain's myelin, leading to symptoms such as seizures and trouble moving.
Zanvastro trials show safety, motor gains
In international trials with 54 patients, Zanvastro helped improve motor skills, especially in younger participants, and met safety standards.
Some patients had mild side effects such as headaches or vomiting.
After years of research, neuropathologist Albee Messing called this approval a huge step forward for families affected by Alexander disease.