FDA Reauthorizes Leucovorin, Seeks Tylenol Label Update

The FDA moved to reapprove and relabel leucovorin (Wellcovorin) for treatment of cerebral folate deficiency, citing a systematic review of studies from 2009–2024 that included patient-level data. The agency and HHS emphasized leucovorin is not a cure and may improve speech-related deficits in only a subset of patients. The announcement, highlighted at a White House event where President Trump and HHS officials promoted the drug, also included an initiative to update acetaminophen (Tylenol) labels to reflect studies suggesting prenatal use could be associated with higher risks of neurodevelopmental conditions. Researchers and public-health experts pushed back, saying the trials supporting leucovorin are small and the evidence linking prenatal acetaminophen to autism is mixed and potentially confounded, and they called for larger, rigorous studies before broader use. Families of autistic children expressed urgency and hope for treatments, while debate continues over regulatory steps, messaging to pregnant people, and the drug’s history — the branded Wellcovorin was withdrawn from sale in 1997 but generic leucovorin has been used off-label for years.