GSK plc confirmed it will submit a supplemental New Drug Application (sNDA) for Wellcovorin (leucovorin) to update the drug's label to include an indication for cerebral folate deficiency (CFD), a rare neurological disorder. The US Food and Drug Administration requested this action as part of its initiative to investigate opportunities for repurposing older medications to treat chronic diseases.
The pharmaceutical company is supporting the FDA's initiative by incorporating data from case reports of genetically confirmed CFD into the US prescribing information for Wellcovorin. GSK does not currently manufacture or market leucovorin, which is available in generic formulations in the United States.
FDA Expected to Approve New Indication
According to agency leaders, the FDA will approve leucovorin as a treatment for children with cerebral folate deficiency and autistic symptoms in the coming weeks. FDA Commissioner Marty Makary, NIH Director Jay Bhattacharya, and Centers for Medicare and Medicaid Services Administrator Mehmet Oz stated that over half of all American children are insured by Medicaid or CHIP, meaning states will be required to cover leucovorin nationwide upon the FDA label change.
The agency leaders contributed to a Politico Magazine opinion piece published September 22, 2025, directing attention toward research suggesting leucovorin could aid children with folate deficiencies. The evidence indicates that leucovorin can assist children diagnosed with autism by helping to improve verbal communications, though the officials emphasized that the drug "is not a cure for autism."
Understanding Cerebral Folate Deficiency
Cerebral folate deficiency is characterized by low concentrations of 5-methyltetrahydrofolate (5-MTHF) - the active form of folate - in the cerebrospinal fluid. This condition may manifest with a range of neuropsychiatric symptoms, making it a significant concern for affected patients and their families.
Scientists have cautioned that current data supporting leucovorin's use in autism patients is limited and requires additional research. However, they acknowledge that this form of vitamin B shows potential in a subset of autism patients.
Leucovorin Background and Current Status
In the United States, leucovorin is currently indicated to diminish toxicity and counteract the effects of impaired methotrexate elimination and inadvertent overdosages of folic acid antagonists. GSK marketed leucovorin under the brand name Wellcovorin from 1983 to 1997, but the drug is now available as a generic medication.
GSK is collaborating with the FDA to expedite the label update process for this medicine. The company's involvement demonstrates the pharmaceutical industry's role in supporting regulatory initiatives to expand treatment options for rare diseases through drug repurposing efforts.
