Overview
- The FDA formalized approval of oral and parenteral leucovorin to treat cerebral folate deficiency with immunological evidence, a biomarker‑defined subset that can co‑occur with autism.
- The decision leans on small studies, including a 12‑week trial of 44 children reporting communication gains, and the agency directed manufacturers to run phase III trials focused on biomarkers, neuroimaging and developmental outcomes.
- President Trump spotlighted the approval in a new autism action plan, while also asserting a link between prenatal acetaminophen use and autism, a claim that has drawn scientific pushback and renewed concerns given HHS Secretary Robert F. Kennedy Jr.’s past discredited vaccine statements.
- Officials said physicians will prescribe and monitor the therapy and that coverage is expected under Medicaid and state insurers, positioning the treatment for broader access.
- Clinicians caution the drug is not a cure and should be reserved for patients confirmed by specialized testing such as CSF 5‑MTHF levels and anti‑FRα antibody assays, with trials to track long‑term safety and benefit.