FDA narrows leucovorin's promise: approved for cerebral folate deficiency, not autism
TL;DR Summary
The FDA approved leucovorin only for cerebral folate deficiency caused by pathogenic FOLR1 variants, not as a treatment for autism, despite earlier pitches by officials that it could help many autistic children. The supporting evidence for autism benefit has been weak and a major trial was retracted due to data concerns. The rare genetic form this approval covers affects a small number of cases, and the agency is encouraging more research to assess any broader autism effects while signaling the treatment’s utility remains limited to the specific cerebral folate deficiency condition.
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