The FDA approved the prescription vitamin leucovorin on Tuesday as the first treatment for cerebral folate deficiency, a rare genetic condition that affects fewer than one in one million people. The approval is significantly narrower than what the Trump administration publicly suggested last September, when President Trump and Health Secretary Robert F. Kennedy Jr. promoted the drug as a potential treatment for hundreds of thousands of children with autism.
What Was Actually Approved
The approval applies specifically to patients with a confirmed variant in the folate receptor 1 gene, known as CFD-FOLR1. This ultrarare genetic condition prevents folate, a key B vitamin, from crossing properly into the brain, causing severe developmental delays, seizures, movement disorders and other neurological complications. Fewer than 50 cases have ever been identified worldwide, according to CNN. The approval covers both generic versions of leucovorin and GSK’s original branded medication, Wellcovorin, though GSK has confirmed it has no plans to relaunch the product. (CNN, FDA.gov)
What Was Not Approved
The FDA declined to approve leucovorin as a broad autism treatment. A senior FDA official told reporters Monday: “We don’t have sufficient data to say that we could establish efficacy for autism more broadly.” FDA reviewers also noted that the largest randomized double-blind, placebo-controlled trial testing the drug in children with autism was recently retracted after errors were identified in the reported data. The American Academy of Pediatrics does not recommend leucovorin’s routine use in children with autism, citing limited evidence. (CNN, NBC News, Fierce Pharma)
The September 2025 Controversy
The gap between Tuesday’s approval and last September’s announcement is significant. At a White House press conference on September 22, 2025, Trump said the leucovorin label change “gives hope to the many parents with autistic children.” FDA Commissioner Marty Makary said the agency was opening the door to the first FDA-recognized treatment for autism and said leucovorin could benefit “hundreds of thousands of kids.” Kennedy called it “an exciting therapy that may benefit large numbers of children.” Following those announcements, leucovorin prescriptions surged by 71% in the weeks that followed, according to a Lancet study published last week, making the drug difficult to find in pharmacies. (CNN, STAT News, Fierce Pharma)
David Mandell, a psychiatry professor at the University of Pennsylvania, called the situation “just terrible for families.” “This is back-and-forth about what treats autism, what doesn’t treat autism, what causes autism, what doesn’t cause it,” Mandell told NBC News. “Families deserve better than that. They deserve more careful science. They deserve more accurate information.” (NBC News)
Autism researcher Alycia Halladay told NBC News that prescriptions are unlikely to fall regardless of Tuesday’s announcement. “The bell has been rung,” she said. “I don’t see that changing with this announcement that it’s only approved for cerebral folate deficiency.” (NBC News)
Why This Matters to You
For families of children with cerebral folate deficiency, Tuesday’s approval is genuinely significant. It is the first FDA-approved treatment for a condition that previously had no recognized therapy.
For the far larger population of families affected by autism, Tuesday’s announcement is a step back from what they were promised. Prescriptions for leucovorin surged after September’s White House briefing, creating shortages of a drug that is now approved for a condition affecting fewer than one in a million people. Families who were told this drug could help their children were acting on information from the highest levels of the US government. They deserve clarity about what the science actually supports.
It is worth thinking about: What responsibility does the federal government have when senior officials publicly promote a drug as a treatment for a condition before the FDA has reviewed the data? With leucovorin prescriptions already surging and the drug in short supply, what happens to patients with the rare genetic condition who actually need it? And with the largest clinical trial on leucovorin and autism recently retracted, what does this mean for the broader question of how the administration communicates about medical research?
-Elijah Iraheta, Editor in Chief, ASC News
