GSK will update Wellcovorin label for cerebral folate deficiency
The U.S. Food and Drug Administration (FDA) is working with GSK to update the label for Wellcovorin (leucovorin). The update could help children and adults with cerebral folate deficiency (CFD), a rare disorder linked to autism-like symptoms.
CFD affects folate transport into the brain, which can lead to developmental delays, seizures, movement problems, and challenges with social communication.
Why this matters for autism
Case studies and research from 2009 to 2024 show that Wellcovorin may improve symptoms in people with CFD, including some features seen in autism. The FDA says it’s part of a bigger effort to repurpose older drugs for chronic conditions.
FDA Commissioner Marty Makary said:
“Children are suffering and deserve access to potential treatments that have shown promise.”
What Wellcovorin does
Leucovorin has long been used in the U.S. to counteract methotrexate toxicity or folic acid overdoses. GSK originally marketed it as Wellcovorin from 1983 to 1997. Today, it’s available as a generic.
Now, the FDA and GSK want to update the label so doctors can safely prescribe it for CFD patients with autism-like challenges.
Next steps
More studies are needed to see how Wellcovorin works for a broader group of patients with autistic features. But the FDA and GSK are moving fast to give families access to the treatment sooner.
George Tidmarsh, Director of the FDA’s Center for Drug Evaluation and Research, said:
“This shows our commitment to finding new uses for existing drugs, including for neurodevelopmental conditions.”
