The U.S. FDA approved , but not for children with the broader diagnosis of autism it promised to help last year.
The U.S. Food and Drug Administration (FDA) has approved leucovorin to treat a rare genetic disorder which causes autism-like symptoms, Reuters reports. However, the drug is not indicated for children with the broader diagnosis of autism, despite expectations voiced in 2025 that approval for this indication was anticipated.
The approval of the drug was based on a systematic review of literature rather than on clinical trial data. The FDA noted that it reviewed 26 reports and reviews up to 2024 concerning the use of the drug. These involved 27 patients who received treatment. Of these, 24 showed improvements in symptoms, including reduction in severity or number of seizures, and improvements in motor function or behavior. Drugs based on the substance are also prescribed to individuals undergoing chemotherapy.
In addition, in March 2026, the FDA approved the drug Tecvayli in combination with Johnson & Johnson’s drug Darzalex.
