FDA Clears Generic Vitamin Drug For Autism Treatment

The Food and Drug Administration has approved leucovorin, a generic medication for a rare brain disorder, which has reportedly shown great promise for people with autism disorder.

FDA Commissioner Dr. Marty Makary said on Tuesday that the approval is “a significant milestone” for patients with the condition.

Leucovorin is an essential form of B vitamin that limits the delivery of folate to the brain. FDA officials estimate the ultrarare condition impacts fewer than 1 in a million people in the U.S.

It is a metabolite of folate that is essential for healthy pregnancies and is recommended for women before conception and during pregnancy.

The current FDA label covers leucovorin’s use in reducing side effects of certain chemotherapy drugs and treating a rare blood disorder.

In September 2025, Makary and US Health and Human Services Secretary Robert F. Kennedy Jr. indicated that the drug would be available to help many more children. Kennedy said they had “identified an exciting therapy that may benefit large numbers of children who suffer from autism.”

It should be recalled that Trump made unsupported claims about the painkiller Tylenol as a cause of autism and cautioned parents about getting too many vaccines for their kids. The President however, noted that changing the label on leucovorin “gives hope to the many parents with autistic children that it may be possible to improve their lives.”

Also, senior FDA officials told reporters Monday that their review was narrowed to focus on the strongest evidence, which only supported the drug’s use by patients with the rare mutation that impacts folate levels in the brain.

The officials also pointed out that one study supporting the drug’s used for autism was retracted earlier this year.

According to reports, Trump officials originally decided to review the drug after speaking with an Arizona-based neurologist who prescribes the drug for autism patients and runs an online education business focused on the experimental treatment.

A paper published in The Lancet last week found that leucovorin prescriptions for children aged five to 17 were 71 per cent higher than normal in the three months immediately following Trump’s late September news conference.