Topic Paragraphs
FDA Approves First Drug to Treat Alexander Disease

FDA Approves First Drug to Treat Alexander Disease

First-ever treatment for rare neurological disorder advances Trump Administration health priorities and offers new hope to patients with no previously approved treatment options.  Alexander disease is a rare, progressive neurological disorder caused by gene mutations that trigger abnormal protein buildup in the brain's supportive cells, damaging the nervous system.

Featured Topics

Recalls
Report a Problem
Warning Letters
Consumer Updates
483s
EUAs
510Ks
Guidance Documents
Drug Databases
Import Program

FDA in Action

Good Clinical Practices Are Not Optional: The FDA’s Commitment to Human Subject Protections and Gold Standard Science in an Era of Global Clinical Research
FDA Approves New Engineered Viral Immunotherapy for Patients with Treatment-Resistant Advanced Melanoma
Supporting and Strengthening Domestic Drug Manufacturing
The FDA at 120: A Long, Distinguished History Protecting Americans
Accelerating and Modernizing Early and Late-Stage Clinical Development
Year 1 Goals in Reducing Animal Testing in Drug Development
Results from Largest-Ever Testing of Infant Formula in the U.S.
FDA PreCheck Pilot Program: Strengthening Domestic Pharmaceutical Manufacturing
Food Chemical Safety Post-Market Assessment Program and Reassessment of BHT, ADA

Press Announcements

September 04, 2026

FDA Takes Steps to Maintain Newborn Access to Life-Saving Starter Nutrition Products

September 04, 2026

FDA Grants Accelerated Approval to a New Breast Cancer Treatment

September 03, 2026

FDA Approves First Drug to Treat Alexander Disease

Products We Regulate

Food
Drugs
Medical Devices
Radiation-Emitting Products
Vaccines, Blood, and Biologics
Animal and Veterinary
Cosmetics
Tobacco Products
Back to Top