The U.S. Food and Drug Administration (FDA) approved leucovorin on Tuesday for treating cerebral folate deficiency, an ultra-rare genetic disorder causing autism-like symptoms. This approval is more limited than previously suggested, as it does not extend to broader autism treatment due to insufficient data.
Key Takeaways
The FDA approved leucovorin for treating cerebral folate deficiency (CFD-FOLR1), an ultra-rare genetic disorder causing autism-like symptoms. The approval is limited to this specific condition and does not extend to broader autism treatment due to insufficient data.
- FDA approves leucovorin specifically for CFD-FOLR1, affecting fewer than 50 people worldwide
- Approval based on systematic review of literature, not clinical trial data
- Leucovorin prescriptions surged by 71% following September press conference promoting it as an autism treatment
- American Academy of Pediatrics does not recommend routine use for autistic children due to lack of evidence
In September, President Donald Trump and Health Secretary Robert F. Kennedy Jr. promoted leucovorin as a potential treatment for autism at a White House press conference. However, the FDA's approval is specifically for cerebral folate deficiency in the receptor 1 gene (CFD-FOLR1), affecting fewer than 50 people worldwide.
The approval was based on a systematic review of published literature rather than clinical trial data, due to the rarity of the disease. The FDA encouraged drugmakers to increase production of leucovorin, as prescriptions surged by 71% following the September press conference. According to the National Organization for Rare Disorders, fewer than 20 people have been reported to have cerebral folate deficiency in scientific literature.
Leucovorin is currently used in chemotherapy patients but can be prescribed off-label for other uses. GSK's Wellcovorin and all generic versions can now include the new indication for cerebral folate deficiency. However, GSK has no plans to manufacture the product again.
The American Academy of Pediatrics does not recommend routine use of leucovorin for autistic children, citing a lack of evidence on benefits. Researchers call for large clinical trials to establish efficacy for broader autism treatment.
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