FDA Stance on Leucovorin and Autism Sparks Debate

In September, the Trump administration announced what it described as “bold actions” targeting autism, highlighting the potential of the generic drug leucovorin as a treatment. Marty Makary, FDA Commissioner, stated in a news release that a “growing body of evidence suggests” the drug might be beneficial. At a White House event, Makary estimated it could aid “20, 40, 50 percent of kids with autism.”

“Hundreds of thousands of kids, in my opinion, will benefit,” Makary commented during the event.

These compelling declarations seemed to have a significant influence. A recent study published in The Lancet revealed that the issuance of new outpatient leucovorin prescriptions for children aged 5 to 17 surged by 71 percent in the three months following the Trump administration’s pronouncements.

However, today it was revealed that the broader FDA did not align with the views of Makary and other administration officials. The regulatory body announced leucovorin's approval was restricted to a rare genetic condition, not for autism.

In statements to the Associated Press, senior FDA officials expressed their conclusion of insufficient evidence to justify expanding the drug’s application to autism. Consequently, their review remained concentrated on treating the rare genetic condition known as cerebral folate deficiency (CFD) in adults, which is caused by a genetic mutation in the folate receptor 1 gene (CFD-FOLR1).