The U.S. FDA approved an expanded indication for leucovorin to treat cerebral folate deficiency linked to FOLR1 variants, but the agency explicitly stopped short of endorsing the vitamin for broad autism treatment. The decision followed months of public discussion after senior administration officials suggested leucovorin could help many children with autism spectrum disorder. FDA statements clarified the approval targets a rare genetic disorder (CFD‑FOLR1) supported by case reports and mechanistic data, not autism generally. The divergence between regulator language and White House comments triggered supply and prescribing shifts and reignited debate about evidence standards for label changes based on real‑world literature versus randomized trials.