• 24 Sep 2025
• Admin
• News Article

GSK to File sNDA for Wellcovorin in Cerebral Folate Deficiency

GSK plc announced plans to submit a supplemental New Drug Application (sNDA) to the US Food and Drug Administration (FDA) seeking a label update for Wellcovorin® (leucovorin). The update would add an indication for the treatment of cerebral folate deficiency (CFD), a rare neurological disorder.

FDA Request to Repurpose Older Drugs

The FDA asked GSK to file the sNDA as part of its initiative to explore new uses for existing medicines in chronic and rare diseases. For Wellcovorin, the FDA identified case reports of genetically confirmed CFD and requested that these data be included in the drug’s prescribing information.

GSK will support the filing as the NDA holder but noted that it does not manufacture or market leucovorin, which is widely available in the US in generic form.

About Cerebral Folate Deficiency

• CFD is defined by low levels of 5-methyltetrahydrofolate (5-MTHF) in cerebrospinal fluid.
• Symptoms can range from developmental delay and movement disorders to psychiatric manifestations.
• Current treatment options are limited, underscoring the potential value of repurposing leucovorin.

Background on Wellcovorin

• In the US, leucovorin is already indicated to:
  - Reduce toxicity and counteract impaired elimination of methotrexate.
  - Treat accidental overdose of folic acid antagonists.
• GSK marketed the branded version, Wellcovorin, between 1983 and 1997.
• Today, leucovorin is only available as a generic therapy.

Next Steps

GSK stated it is working closely with the FDA to secure a label update swiftly, in line with the agency’s push to repurpose older medicines for unmet medical needs.

"Our goal is to align with FDA on expanding the clinical utility of leucovorin where it may benefit patients with rare disorders like CFD," the company noted.