FDA Approves Drug for Disorder That Causes Autism-Like Symptoms

The Food and Drug Administration on March 10 approved a long-available generic drug called leucovorin for use against a rare disorder that causes autism-like symptoms.

Regulators approved the drug for treating cerebral folate deficiency in children and adults who have a confirmed variant in the folate receptor 1 gene (FOLR1), based on a systemic review of studies, rather than clinical trial data.

“Today’s approval represents a significant milestone for patients living with cerebral folate transport deficiency due to the FOLR1 variant, a rare genetic condition that has had no FDA-approved treatment options until today,” Dr. Marty Makary, the FDA’s commissioner, said in a statement. “This action may benefit some individuals with FOLR1-related cerebral folate transport deficiency who have developmental delays with autistic features.”

Dr. Tracy Beth Hoeg, acting director of the FDA’s Center for Drug Evaluation and Research, said that the approval “demonstrates the FDA’s commitment to rapidly identifying effective treatments for ultra rare diseases while maintaining the same evidentiary standards for approval.”

Leucovirin, also known as folonic acid, has for years been given to cancer patients to prevent side effects from chemotherapy. It has been used by a growing number of doctors for autistic patients.

“For those with impaired brain folate transport, it’s a logical, evidence-based option that’s delivering real clinical gains in autism and other conditions,” Dr. David Danish, a psychiatrist and president of Philadelphia Integrative Psychiatry, told The Epoch Times previously in an email.

In September 2025, Makary said that leucovorin could help up to 50 percent of kids with autism, while Health Secretary Robert F. Kennedy Jr. said that the drug “may benefit large numbers of children who suffer from autism.”

Outpatient prescriptions for leucovorin among children aged 5 to 17 skyrocketed in the wake of that briefing, according to a study published on March 5. Doctors can prescribe FDA-approved drugs for other purposes, a practice known as off-label.

The new approval is much narrower than officials had suggested.

“While we did originally approach this with a broad scope, as we looked through the scientific data, we ended up narrowing our scope to come to the strongest data evidence that we could find to support the approval,” a senior U.S. Food and Drug Administration official, speaking on condition of anonymity, told reporters on a call on Tuesday.

Wider approval would have been based on research that indicates many children have autoantibodies that block folate, a vitamin important for brain signaling, from entering the brain, causing a deficiency similar to cerebral folate deficiency.

“Right now, we don’t have sufficient data to say that we could establish efficacy for autism more broadly,” the FDA official said.

A spokesperson for GlaxoSmithKline, which holds the drug application for leucovorin and worked with the FDA to update the drug’s labeling, told The Epoch Times in an email that it will not be producing the drug.

The expanded approval allows all generic versions of the drug to include the new indication, according to the FDA, which is encouraging drugmakers who produce it to increase production.

The American Academy of Pediatrics, which partners with GlaxoSmithKline and other pharmaceutical companies, says that leucovorin should only be prescribed to patients with cerebral folate deficiency after going over the available evidence and the drug’s side effects, which include nausea and skin issues.

Reuters contributed to this report.