<2> FDA Approves Leucovorin for Rare Genetic Condition, But Not for Autism
<3> FDA Authorizes Treatment for FOLR1 Related Cerebral Folate Transport Deficiency
The US Food and Drug Administration (FDA) has granted approval for the expanded use of leucovorin (Wellcovorin) to treat adults and children with FOLR1 related cerebral folate transport deficiency. This marks the first FDA-authorized treatment for the rare genetic condition. However, the FDA’s decision also came with a surprise twist, as it did not approve the drug for autism-related symptoms, despite the hype generated by the Trump administration in September 2025.
< href='https://who.int/news-room/q-and-a/detail/what-is-folate-deficiency' target='_blank'>Folate deficiency symptoms and effects are often associated with the condition, but the FDA has stated that there is not sufficient data to support the use of leucovorin as an autism treatment. According to anonymous FDA officials quoted by CNN, the agency does not have the evidence required for a broader approval.
<3> FDA’s Decision: A Step Back from Autism Treatment Claims
The FDA’s decision to step back from its previous claims about