FDA just cleared three breakthrough drugs—for rare neurological disease, ADHD, and breast cancer

In early September 2026, the U.S. Food and Drug Administration approved three distinct therapies, each representing a regulatory or scientific first in its category. On September 3, 2026, the FDA approved Zanvastro (zilganersen), developed by Ionis Pharmaceuticals, for Alexander disease (AxD) in both pediatric and adult patients.