FDA Approves Leucovorin for Rare Genetic Disorder, Walks Back Autism Claims

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The Food and Drug Administration has approved the generic drug leucovorin for a rare genetic condition that limits folate delivery to the brain, walking back previous claims by the Trump administration that the drug showed great promise for treating autism. The FDA said its review focused on the strongest evidence, which only supported the drug's use for the rare genetic disorder, not autism more broadly.

Why it matters

This case highlights the challenges in developing effective treatments for complex neurological conditions like autism, where scientific evidence often lags behind political rhetoric and patient demand. It also raises questions about the FDA's role in managing public expectations around experimental therapies.

The details

The FDA approved leucovorin, a folate metabolite, for patients with a rare genetic condition that causes movement disorders, seizures and other neurological problems that can resemble autism symptoms. However, the agency walked back previous statements made by President Trump and other officials that the drug showed great promise for treating autism more broadly. The FDA said its review focused on the strongest evidence, which only supported the drug's use for the rare genetic disorder, not autism. Professional medical societies also say it's unclear whether leucovorin helps people with autism.

• The FDA approved leucovorin on Tuesday, March 10, 2026.
• In September 2025, President Trump and FDA Commissioner Marty Makary announced the drug was under review to benefit patients with autism.
• Earlier in 2026, a study supporting the drug's use for autism was retracted.

What’s next

FDA officials said the agency is allowing imports of the drug by foreign drugmakers to help boost supply, as the original manufacturer GSK does not plan to relaunch its version.