Fortress Biotech and Cyprium Therapeutics Announce U.S. FDA Approval of ZYCUBO® (copper histidinate), the First and Only Approved Treatment for Menkes Disease in the United States

Core Insights - The FDA has approved ZYCUBO (copper histidinate) for the treatment of Menkes disease in pediatric patients, marking it as the first and only FDA-approved treatment for this condition [1][4][9] - Cyprium Therapeutics, a subsidiary of Fortress Biotech, will receive a Rare Pediatric Disease Priority Review Voucher (PRV) and is eligible for tiered royalties and up to $129 million in development and sales milestones from Sentynl Therapeutics [2][4] Company Developments - Fortress Biotech and Cyprium Therapeutics announced the FDA approval of ZYCUBO, which is a significant milestone for both the company and patients suffering from Menkes disease [4] - The approval of ZYCUBO is supported by positive clinical efficacy results, showing a nearly 80% reduction in the risk of death for patients receiving early treatment compared to an untreated control cohort [4][9] Product Information - ZYCUBO is a subcutaneous injectable formulation that restores copper homeostasis in patients with Menkes disease, a rare genetic disorder caused by mutations in the ATP7A gene [3][9] - The drug has received multiple designations from the FDA, including Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designation [6] Market Context - Menkes disease has a minimum birth prevalence of 1 in 34,810 live male births, with potentially higher rates based on recent studies [7][8] - The condition is characterized by severe neurological symptoms and high mortality rates in untreated cases, emphasizing the need for effective treatments like ZYCUBO [8]