FDA doesn’t approve leucovorin for autism. What can it be used for?

The FDA narrowed leucovorin's use to a rare disorder, a major shift after Trump and RFK Jr. promoted it for autism.

The Food and Drug Administration has sharply narrowed the approved uses of leucovorin, limiting the drug to a rare genetic disorder, a significant pullback after President Donald Trump and Health and Human Services Secretary Robert F. Kennedy Jr. promoted it as a promising autism treatment.

The agency said on Tuesday, March 10, that leucovorin is now approved only for patients with cerebral folate deficiency, a rare disorder that can include autistic features. The decision marks a major shift from the broader framing offered by the Trump administration last fall, when officials touted the drug as a possible way to address autism despite a lack of scientific consensus supporting such claims.

At a White House briefing in September, Trump, Kennedy, and other officials outlined plans to find what they called an "answer to autism," linking rates to Tylenol use in pregnancy, without evidence, and touting leucovorin as a way to treat autism symptoms.

The administration has repeated claims about its plans to find the "cause" for autism. Decades of medical and scientific consensus indicate there is no singular source, including medication, that can be attributed to autism, which is probably the result of multiple contributing factors, including genetics.

Trump said in September that changing the label on leucovorin would give "hope to the many parents with autistic children that it may be possible to improve their lives.” And, while the new approval does mention "autistic features," it applies to a remarkably smaller demographic than originally presented months ago.