Brain HealthFDA Approves Leucovorin for Cerebral Folate Deficiency Caused by FOLR1 Variants
The FDA has approved Wellcovorin (leucovorin calcium) tablets as the first treatment specifically indicated for cerebral folate deficiency in patients with confirmed FOLR1 gene variants. Cerebral folate deficiency occurs when the folate receptor alpha protein — encoded by FOLR1 — fails to transport folate across the blood-brain barrier efficiently, starving the brain of a nutrient critical for neurodevelopment and neurological function. This approval covers both adult and pediatric patients. Leucovorin is a reduced form of folate that can partially bypass the defective transport mechanism, potentially restoring adequate folate levels in the central nervous system. The approval marks a significant milestone for patients and families affected by this rare but serious condition, which can cause developmental delays, movement disorders, seizures, and cognitive decline. It also highlights the growing recognition of rare metabolic neurological diseases as actionable therapeutic targets.
