Particle.news
Download on the App Store
16 ARTICLES
·
1h ago

FDA Backs Leucovorin Label Update for Cerebral Folate Deficiency Linked to Autism Symptoms

Experts call the evidence preliminary following officials' push for Medicaid coverage.

Image
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.
Medicare and Medicaid Administrator Mehmet Oz, right, and President Donald Trump speak at an event in the East Room of the White House in Washington, D.C on July 30, 2025.

Overview

  • An FDA Federal Register notice reinstated a GSK leucovorin application and cited literature and case reports in roughly 40 cerebral folate deficiency patients, with the agency urging a label update.
  • GSK said it will file a supplemental application to add cerebral folate deficiency to Wellcovorin’s label, a step HHS says could enable state Medicaid coverage tied to autism-related care.
  • HHS and FDA officials highlighted potential benefits for speech-related deficits in a subset of children, and the NIH plans to fund additional studies on safety and effectiveness.
  • Researchers and advocacy groups describe the clinical evidence as limited to small, heterogeneous trials, recommending targeted use based on testing for folate-transport abnormalities rather than broad prescribing.
  • President Trump promoted unproven links between acetaminophen and autism without new data, while HHS emphasized cautious language, and CMS chief Mehmet Oz pledged to divest after concerns over ties to a supplement retailer selling folinic acid.