FDA contradicts Trump admin, declines to approve generic drug for autism
Source: Ars Technica
Background
In September, the Trump administration announced “bold actions” on autism, touting the generic drug leucovorin as a promising treatment. In a news release, Marty Makary, commissioner of the Food and Drug Administration, claimed a “growing body of evidence” suggested the drug could be helpful. At a White House press event, Makary suggested it might help “20, 40, 50 percent of kids with autism.”
“Hundreds of thousands of kids, in my opinion, will benefit,” he added during the event.
Study Findings
A study published in The Lancet last week reported that new outpatient prescriptions of leucovorin for children ages 5 to 17 increased by 71 percent in the three months following the administration’s actions.
FDA Response
The FDA later clarified its position, stating it had approved leucovorin for a rare genetic condition—cerebral folate deficiency (CFD) in adults caused by a mutation in the folate receptor 1 gene (CFD‑FOLR1)—but not for autism.
In comments to the Associated Press, senior FDA officials said they found little evidence for expanding the drug’s use to autism and therefore limited its review to the treatment of the rare genetic condition.
- FDA approval announcement: https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-cerebral-folate-transport-deficiency?utm_medium=email&utm_source=govdelivery
- AP report on FDA stance: https://apnews.com/article/autism-fda-drug-leucovorin-trump-kennedy-97a1b5092de76b7956f778f374189a0c