health
March 10, 2026
FDA Approves Leucovorin as First Drug for Rare Genetic Disorder, After Touted as Autism Treatment
The move comes months after the Trump administration touted leucovorin as a potential therapy for a broader group of patients with autism symptoms.
TL;DR
- Leucovorin, a prescription vitamin, is approved as the first treatment for cerebral folate deficiency.
- The FDA found sufficient data to approve leucovorin for cerebral folate deficiency, a rare genetic disorder.
- The agency stated there was not enough data to establish efficacy for autism spectrum disorder more broadly.
- Cerebral folate deficiency shares overlapping features with autism and can cause severe developmental delays.
- The approval was based on a systematic review of published literature, not a randomized controlled trial.
- The FDA is encouraging manufacturers to increase production of leucovorin.
- GSK, the original marketer of the drug, has no current plans to relaunch it.
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