The FDA has approved the prescription drug for cerebral folate deficiency, a rare neurological disorder, not for autism. The decision clarifies months of public debate after federal officials previously promoted the drug as a possible autism treatment.
The FDA said there is not enough evidence to support leucovorin as a treatment for autism. Instead, the approval applies to cerebral folate deficiency, a condition linked to impaired folate transport to the brain and associated with serious neurological symptoms.
Cerebral folate deficiency is extremely rare, affecting fewer than one in 1 million people in the United States, according to reported coverage of the FDA decision. Symptoms can include developmental delay, seizures, movement problems, and cognitive decline.
FDA Leucovorin Approval Explained for Cerebral Folate Deficiency
The announcement also marks a shift from earlier messaging by Trump administration officials, who had highlighted leucovorin as a potential breakthrough for autistic children. Since then, senior officials have said the available evidence does not support that broader use.
A recent Lancet report found a 71% increase in pediatric leucovorin prescriptions after the White House briefing in September 2025. That surge added urgency to the FDA’s effort to clarify what the drug is approved to treat, and is not.
Leucovorin, also known as folinic acid, has already been used in other medical settings for years. However, experts quoted in coverage of the new approval say cerebral folate deficiency should not be treated as the same condition as autism, even if some symptoms can overlap.
