Overview

• The FDA published a Federal Register notice reapproving GSK’s leucovorin and signaling use for cerebral folate deficiency with continued treatment in autistic children who show language, social or adaptive gains.
• GSK, which still holds the drug’s New Drug Application, said it will submit a label‑update application at the FDA’s request, and the NIH plans to monitor outcomes and study broader effects.
• Health officials said Medicaid and CHIP would be required to cover the therapy once the label is updated, creating a new access pathway for eligible patients.
• Researchers and advocacy groups say current support comes from a handful of small, heterogeneous trials, leaving key questions about who benefits, appropriate dosing and pediatric safety.
• A White House event promoting the drug also featured unproven claims about Tylenol and autism, prompting scientific pushback and a reaffirmation from the UK’s MHRA that paracetamol in pregnancy has not been shown to cause autism.