
FDA approves leucovorin as first drug for rare genetic disorder, after touting it as autism treatment
CNBC
The move comes months after the Trump administration touted leucovorin as a potential therapy for a broader group of patients with autism symptoms.
The Food and Drug Administration on Tuesday approved a decades-old prescription vitamin called leucovorin as the first treatment for a rare genetic disorder in certain adults and children.
The move comes months after the Trump administration touted leucovorin as a potential therapy for a broader group of patients with autism spectrum disorder symptoms. The claim sparked skepticism among some in the medical and research community, but fueled excitement among families, spiking prescriptions of the drug in the U.S.
One FDA official told reporters Monday that "we don't have sufficient data to say that we could establish efficacy for autism more broadly" but said the agency is open to interest from companies in studying leucovorin in the autism population.













