Summary
The FDA approved leucovorin for a rare genetic disorder affecting folate delivery to the brain. Although previously discussed as a potential treatment for autism by President Trump, the FDA found insufficient evidence to support its use for autism. The drug's approval is specifically for those with the rare disorder, not for autism treatment.
Key Facts
- The FDA approved leucovorin for a rare brain disorder affecting folate levels in less than 1 in a million people in the U.S.
- President Trump previously mentioned leucovorin as promising for children with autism.
- FDA review found little evidence supporting leucovorin for autism.
- A study claiming benefits for autism was retracted earlier this year.
- Leucovorin is known for reducing side effects of chemotherapy and treating a rare blood disorder.
- Some small studies show possible benefits for certain autism patients, but medical societies remain cautious.
- Leucovorin prescriptions for children rose following Trump's announcement.
- The FDA is allowing imports to increase drug supply due to a prescription increase.
