FDA backs away from RFK Jr.’s claims about drug’s promise for autism patients

Food and Drug Administration Commissioner Marty Makary said in September he was changing leucovorin’s label because it could help “hundreds of thousands” of children with the neurological condition.

FDA Commissioner Marty Makary speaks alongside President Donald Trump and HHS Secretary Robert F. Kennedy Jr. during announcements on autism in the Roosevelt Room at the White House on Sept. 22, 2025. | Francis Chung/POLITICO

The Food and Drug Administration does not have sufficient evidence to back the use of the generic drug leucovorin for treating children with autism, two senior agency officials told reporters Monday.

The agency’s announcement represents a major change in its view since Health Secretary Robert F. Kennedy Jr. and FDA Commissioner Marty Makary touted the drug, also known as folinic acid, as a promising therapy that could benefit some children with autism at a September news conference with President Donald Trump.

Then, Makary had said the drug would help “hundreds of thousands” of kids and that one study had shown that among kids with autism and chronic folate deficiency, “two-thirds of kids with autism symptoms had improvement and some marked improvement.” Folate deficiency doesn’t allow folate, an important vitamin for the brain, to get into the brain cells, Makary explained at the time.

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