Overview
- FDA officials said randomized trials were not feasible for the disorder, which they estimate affects fewer than 1 in a million people in the U.S.
- Tuesday’s action allows generic labels to add the new indication, with the agency urging manufacturers to boost output and permitting imports to bolster supply.
- GSK does not plan to relaunch its branded version, though leucovorin remains widely available in generic form for its existing approved uses.
- A September White House event promoting leucovorin for autism preceded a 71% rise in pediatric prescriptions reported by The Lancet and contributed to recent shortages.
- One autism trial supporting leucovorin was retracted this year, and groups such as the American Academy of Pediatrics do not recommend routine use for autism due to limited data.