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1h ago

FDA Moves to Update Leucovorin Label for Cerebral Folate Deficiency, Citing Potential Speech Benefits in Some Autistic Children

Experts call the evidence preliminary, urging larger trials with targeted testing before clinicians consider folate therapy for autism.

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Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo
President Donald Trump makes an announcement about autism with National Institutes of Health Director Jay Bhattacharya, Food and Drug Administration Commissioner Marty Makary, Secretary of Health and Human Services Robert Kennedy Jr., and Medicare Services Administration Mehmet Oz in the Roosevelt Room of the White House, in Washington on Sept. 22, 2025.
Signage is pictured in the main lobby of GSK offices in London, Britain, February 20, 2025. REUTERS/Chris J. Ratcliffe/ File Photo

Overview

  • The FDA published a Federal Register notice approving a GlaxoSmithKline formulation of leucovorin and cited data from 40 patients with cerebral folate deficiency.
  • HHS and the FDA plan a label update to recognize treatment of cerebral folate deficiency and to note leucovorin as a potential option for speech‑related deficits in a subset of autistic patients, a change that could enable Medicaid coverage.
  • NIH leaders signaled new research initiatives to assess leucovorin’s safety and efficacy for autism‑related symptoms.
  • Autism Science Foundation and leading researchers say current support comes from small, heterogeneous studies and urge larger, well‑controlled trials.
  • Clinicians recommend testing for folate transport issues before prescribing and warn of possible side effects such as irritability or aggression, as the White House promoted the move and advanced an unproven claim linking acetaminophen use in pregnancy to autism.