FDA finds little evidence that a generic drug could help many people with autism

Associated Press (AP)EN 3 min read 100% complete by By MATTHEW PERRONEMarch 10, 2026 at 02:23 PM

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The FDA approved a generic version of leucovorin for a rare genetic condition affecting folate delivery to the brain, impacting fewer than 1 in a million people in the U.S. This action contrasts with previous suggestions by President Trump and FDA Commissioner Makary that the drug could benefit a significant percentage of individuals with autism. The FDA clarified that its review focused on evidence supporting the drug's use for the rare folate deficiency, despite earlier claims about its potential for broader autism treatment. Officials noted that a study supporting leucovorin's use for autism was retracted. Medical societies, including the American Academy of Pediatrics, do not recommend routine use of leucovorin for autistic children, even those with cerebral folate deficiency, due to limited evidence of its effectiveness.