On September 22, 2025, the U.S. Food and Drug Administration (FDA) authorized leucovorin, a folic acid derivative, to treat autism symptoms.
The approval, announced in the Federal Register, aligns with President Donald Trump’s plan to discuss the drug at a White House event.
Drug History
Leucovorin, once sold as Wellcovorin by GSK, was pulled from the market for non-safety reasons. Its new approval targets a fresh medical use.
Clinical Support
Data from over 40 patients, including kids and adults, showed leucovorin helps with cerebral folate deficiency (CFD), a condition tied to autism-like and neuropsychiatric symptoms, per the Federal Register.
Health Impact
This approval offers new hope for managing autism symptoms linked to CFD, marking a significant step in treatment options based on patient studies.
