US FDA Approves 46-Year-Old Drug Leucovorin for Rare Childhood Genetic Disease
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US FDA Approves 46-Year-Old Drug Leucovorin for Rare Childhood Disease
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CNBC Awaaz•10-03-2026, 20:02
US FDA Approves 46-Year-Old Drug Leucovorin for Rare Childhood Disease
•The US FDA has approved the 46-year-old drug Leucovorin for treating Cerebral Folate Deficiency, a rare genetic disease in children.
•Cerebral Folate Deficiency prevents essential folate (Vitamin B9) from reaching the brain, impacting development and causing symptoms similar to autism in children under two.
•Leucovorin, also known as folinic acid, is a synthetic Vitamin B9 previously used in cancer therapy; this approval marks a new medical application.
•Despite some discussions linking it to autism treatment, the FDA clarified that approval is strictly for Cerebral Folate Deficiency due to insufficient data for widespread autism use.
•The approval was based on a review of published studies and case reports, not large trials, demonstrating a strong effect; FDA anticipates increased demand and urges companies to boost production.
