FDA Approves Zycubo (copper histidinate) for the Treatment of Menkes Disease in Paediatric Patients – Cyprium Therapeutics

Fortress Biotech, Inc. announced that the U.S. Food and Drug Administration has approved ZYCUBO (copper histidinate, formerly known as CUTX-101) for the treatment of Menkes disease in pediatric patients.

The approval is supported by positive topline clinical efficacy results for ZYCUBO, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment (“ET”) with ZYCUBO, compared to an untreated contemporaneous external control (“EC”) cohort, with a nearly 80% reduction in the risk of death. Median overall survival (“OS”) was 177.1 months for ZYCUBO ET cohort compared to 17.6 months for the EC cohort. The most common adverse reactions (incidence ≥7%) were pneumonia, viral infection, respiratory failure, seizure, bacterial infection, hemorrhage, hypotension, vomiting, tachycardia, pyrexia, volume depletion, fracture, dyspnea, transaminases elevation, diarrhea, fungal infection, anemia, and local administration reaction. ZYCUBO has received Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designation from the FDA. Copper histidinate has also been granted Orphan Designation by the European Medicines Agency.