United States of America

FDA Approves Leucovorin for Rare Genetic Brain Disorder

Is the FDA's approval of leucovorin a milestone for patients or a debacle following unproven autism claims?

MAR 11·Updated MAR 20

FDA Approves Leucovorin for Rare Genetic Brain Disorder

Story

Above: The FDA's White Oak campus in Silver Spring, Maryland, on July 17, 2025. Image credit: ISSAM AHMED/AFP /Getty Images

The Facts

The Food and Drug Administration (FDA) approved the prescription vitamin leucovorin on Tuesday as the first treatment option of its kind for adults and children suffering from cerebral folate deficiency with a confirmed variant in the folate receptor 1 gene.
U.S. Food and Drug Administration
CNBC
The Epoch Times
Cerebral folate deficiency is a rare genetic condition estimated to affect fewer than one in 1 million people that disrupts folate transport to the brain, causing serious neurological complaints, including movement disorders, seizures and developmental delays.
NCBI
U.S. Food and Drug Administration
The FDA based its approval on a systematic review of published literature and patient case reports rather than randomized controlled trials, which officials claimed would be potentially unethical given the "dramatic" treatment response seen in children with the genetic condition.
U.S. Food and Drug Administration
CNN
It follows claims made by Health and Human Services Secretary Robert F. Kennedy Jr. and FDA Commissioner Dr. Marty Makary in September that leucovorin was a promising treatment for autism, with Kennedy describing it as an "exciting therapy" during a televised briefing.
Youtube
Youtube
Politico
Donald Trump
Following that announcement, new leucovorin prescriptions for children aged 5 to 17 in the U.S. more than doubled between Sept. 22 and Dec. 7, 2025, rising 113% above the expected figure, according to a report published in The Lancet.
The Lancet
FDA officials, however, backed away from the link between leucovorin and autism on Tuesday, with one telling reporters that the federal agency did not have "sufficient data" to "establish efficacy for autism more broadly."
The Hill
Politico
National Post

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The Spin

Pro-establishment narrative

The FDA's approval of leucovorin for cerebral folate deficiency marks a significant milestone for patients with this rare genetic condition who previously had no treatment options to turn to. The groundbreaking decision is yet another example of the FDA's commitment to addressing the critical needs of individuals who are suffering from severe neurological complications.

U.S. Food and Drug Administration

Establishment-critical narrative

The administration's handling of leucovorin has been a total debacle. After hyping it as a potential autism treatment on live television, the administration has quietly walked back its claims after admitting it lacks the evidence to support its observations. In the future, perhaps the White House should make announcements after obtaining the proof rather than before.