FDA approves leucovorin for cerebral folate deficiency, not for autism
The Facts
- The FDA approved leucovorin for cerebral folate deficiency (including a FOLR1-related form described as a rare/ultra-rare genetic condition), not for autism broadly.
- FDA officials said there is insufficient evidence/data to establish leucovorin’s efficacy for autism more broadly.
- The approval represents a pullback from September statements by President Donald Trump and senior health officials, including FDA Commissioner Marty Makary, who had promoted leucovorin as promising for autism symptoms and suggested it could help many children.
- FDA officials estimate the approved condition affects fewer than 1 in a million people in the United States.
- Leucovorin is a decades-old drug and has been used for other indications, including counteracting toxic effects of certain cancer drugs such as methotrexate.
- The FDA’s decision to approve leucovorin for the rare folate-transport/folate-deficiency disorder was based on a review of published literature rather than a new clinical trial.
Context
What exactly did the FDA approve leucovorin to treat?
The FDA approved leucovorin for cerebral folate deficiency—a rare genetic disorder involving impaired delivery/transport of folate (vitamin B9) to the brain, which can produce developmental delays and autism-like features. Reuters,PBS.org
Did the FDA approve leucovorin as a treatment for autism symptoms?
No. FDA officials said the agency does not have sufficient data to establish efficacy for autism more broadly, and the approval was limited to the rare cerebral folate deficiency indication. NBC News,Reuters,Washington Post
Why did the FDA narrow the scope compared with earlier public claims?
A senior FDA official said that while the agency initially approached the review with a broader scope, it narrowed the approval to where the scientific evidence was strongest, citing a lack of data for autism broadly. Reuters,Reuters
The receipts — all 89 sources
Wire services (23)
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Independent coverage (50)
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La RevistaLa Revista
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Washington ExaminerFDA declines to approve leucovorin for autism
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