The US Food and Drug Administration (FDA) has granted its approval for the expanded use of GSK’s Wellcovorin (leucovorin calcium) tablets to treat cerebral folate deficiency (CFD) in patients with a confirmed variant in the folate receptor one (FOLR1) gene.
The approval marks the first authorised treatment for cerebral folate deficiency, a rare genetic neurological disorder affecting brain folate transport.
It followed a systematic review of published literature, including case reports with patient-level data and mechanistic studies.
CFD leads to seizures, movement disorders, developmental delays, and neurological complications. Individuals with FOLR1-related cerebral folate transport deficiency (CFD-FOLR1) have had limited options until now.
FDA worked with GSK, the new drug application (NDA) holder for Wellcovorin, to update product labelling and ensure the inclusion of essential scientific information required for safe and effective drug use in adult and paediatric CFD-FOLR1 patients.
FDA commissioner Marty Makary said: “Today's approval represents a significant milestone for patients living with cerebral folate transport deficiency due to the FOLR1 variant, a rare genetic condition that has had no FDA-approved treatment options until today.
"This action may benefit some individuals with FOLR1-related cerebral folate transport deficiency who have developmental delays with autistic features.”
FDA Center for Drug Evaluation and Research acting director Tracy Beth Hoeg said: “The approval of leucovorin for CFD-FOLR1 demonstrates the FDA’s commitment to rapidly identifying effective treatments for ultra-rare diseases while maintaining the same evidentiary standards for approval.
“It also provides a good example of how observational or ‘real world’ evidence can lead to an FDA approval when the product is shown to provide a clear clinical benefit compared with what is seen with the natural history of the disease.”
Before this, the FDA updated its regulatory framework for biosimilar medicines to support reductions in domestic drug prices. With the introduction of new draft guidance, the regulator relaxed certain regulations surrounding biosimilar development, aiming to streamline and simplify the approval process.
