FDA Approves Folinic acid for Rare Disorder, Not Autism

The United States===Food and Drug Administration (FDA) has approved leucovorin for cerebral folate deficiency, a rare genetic disorder affecting less than one in a million people. This approval comes after earlier public statements by Donald Trump, Marty Makary, and Robert F. Kennedy Jr. suggested a broader potential for leucovorin as an autism treatment, leading to a significant increase in prescriptions. However, the FDA explicitly clarified that the approval does not extend to autism due to insufficient supporting data, including a retracted randomized trial. This decision has caused disappointment among families who had hoped for an FDA-recognized autism treatment and raised concerns about future access and insurance coverage for leucovorin. Organizations like the Autism Science Foundation and the American Academy of Pediatrics have also noted the lack of evidence for leucovorin's efficacy and safety for most people with autism.