FDA: Leucovorin Lacks Autism Treatment Efficacy
FDA found leucovorin lacks evidence for autism treatment, reinforcing its stance on evidence-based medicine and guiding healthcare away from unproven…
Ayla Demirhan ·
The U.S. Food and Drug Administration (FDA) has concluded that there is insufficient scientific evidence to support the effectiveness of leucovorin in treating autism spectrum disorder. This determination, announced on Tuesday, March 12, 2026, follows a comprehensive review of available scientific literature and clinical data concerning the drug's application for autism.
The regulatory body's assessment found no established benefits for autism patients, despite some advocacy for its use in this context. Leucovorin, a generic form of folic acid, is currently approved for specific medical conditions, including certain cancer treatments and as an antidote for methotrexate overdose. However, its efficacy for autism has not met the FDA's rigorous standards for new indications.
### Regulatory Review Process
### Regulatory Review Process
The FDA's decision underscores its commitment to evidence-based medicine. The agency's review process typically involves evaluating data from controlled clinical trials and peer-reviewed studies to ascertain a drug's safety and effectiveness for a particular use. In this instance, the data presented did not demonstrate a clear therapeutic benefit for individuals with autism spectrum disorder.