MedPage Today September 23, 2025
Evidence supporting the drug’s use in autism patients is limited
The FDA said on Monday that it will relabel leucovorin calcium tablets (Wellcovorin) as a treatment for adults and children with cerebral folate deficiency, a rare condition affecting folate transport into the brain that has been associated with developmental delays that include autistic features in some people.
The drug, also known as folinic acid, is a type of folate used with cancer drugs like methotrexate to counteract their toxic effects. Leucovorin can cross into the brain even when folate transport is impaired.
The FDA’s decision was based on a systematic analysis of literature published between 2009 and 2024 that suggested leucovorin may improve symptoms of cerebral folate deficiency.
“Published case...
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