The U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for CUTX-101 (Copper Histidinate), a treatment for Menkes disease, a rare X-linked recessive pediatric disease caused by mutations in the copper transporter ATP7A gene. The NDA has been granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2025.
CUTX-101 has demonstrated significant clinical efficacy, with early treatment showing a nearly 80% reduction in the risk of death compared to an untreated historical control cohort. The median overall survival was 177.1 months for the CUTX-101 early treatment cohort, versus 16.1 months for the untreated cohort. The drug has previously received FDA Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designations, as well as Orphan Drug Designation from the European Medicines Agency.
Menkes disease is a severe condition with no known cure or FDA-approved treatments, typically resulting in death between 2 to 3 years of age. The disease affects copper transport to various organs and systems, leading to severe neurological symptoms and connective tissue problems.
Sentynl Therapeutics, Inc., a U.S.-based biopharmaceutical company, has assumed responsibility for the development and commercialization of CUTX-101. Cyprium Therapeutics, the original developer, is eligible for royalties and retains ownership of any Priority Review Voucher that may be issued upon NDA approval.
This development represents a significant milestone for patients suffering from Menkes disease and their families, offering hope for the first FDA-approved treatment for this devastating condition.
