FDA approves leucovorin for rare genetic condition, but not for autism | CNN

The prescription drug leucovorin is getting a label update, but it’s not what that the US Food and Drug Administration suggested during a White House briefing in September, when officials touted the drug as a potential treatment for thousands of children with autism. On Tuesday, the FDA approved the high-dose B vitamin — a treatment long used to counteract the side effects of chemotherapy — for cerebral folate deficiency in the receptor 1 gene, a genetic condition that’s estimated to affect only about 1 in 1 million people. Fewer than 50 cases have ever been identified worldwide. FDA Commissioner Dr. Marty Makary called Tuesday’s approval “a significant milestone” for patients with the condition. But in September, Makary and US Health and Human Services Secretary Robert F. Kennedy Jr. indicated that the drug would be available to help many more children. Standing alongside President Donald Trump at a White House briefing, Kennedy said they had “identified an exciting therapy that may benefit large numbers of children who suffer from autism.” The update on leucovorin may hit some families of children with autism especially hard. At the September briefing — when T

Mar 10, 2026 → Mar 10, 2026