The U.S. Food and Drug Administration today approved the Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients.
FDA Approves First Treatment for Children With Menkes Disease
FDA approved Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients. Menkes disease is a genetic copper absorption defect causing neurodegeneration; Zycubo provides copper in a form that bypasses the intestinal defect. In two open-label trials, 66 treated patients were followed up to three years and compared with 17 untreated…
breakthrough therapy clinical trial clinical trials copper histidinate copper replacement therapy Drug approval drug development fast track FDA FDA approval Menkes disease orphan designation orphan disease orphan drug Orphan Drug Designation pediatric pediatrics priority review rare disease Sentynl Therapeutics subcutaneous injection ultra-rare disease Zycubo