FDA approves leucovorin for rare genetic disorder but not for autism
Mar 11th 2026
After White House promotion of leucovorin as a potential autism treatment, the FDA approved the drug only for a rare FOLR1-related folate disorder in adults and said evidence does not support expanding its use to autism.
- FDA approved leucovorin for adults with cerebral folate deficiency caused by FOLR1 mutations, not for autism.
- Senior FDA officials said there is little evidence supporting leucovorin as an autism treatment and narrowed the drug review accordingly.
- The Trump administration publicly promoted leucovorin as a promising autism treatment at a White House event, with claims it could help a substantial share of children.
- A Lancet-linked study found outpatient prescriptions for leucovorin in children ages 5 to 17 rose 71 percent in the three months after the administration's actions.