Sentynl Therapeutics Announces U.S. FDA Acceptance and Priority Review of New Drug Application for CUTX-101 (Copper Histidinate) Product Candidate for Treatment of Menkes Disease

Core Insights - The FDA has granted a six-month priority review for CUTX-101, a potential first treatment for Menkes disease, with a target action date of June 30, 2025 [1][3] - CUTX-101 has shown promising clinical efficacy, with nearly 80% reduction in the risk of death for patients receiving early treatment compared to untreated controls [2][6] - Menkes disease is a rare and fatal pediatric condition with a prevalence estimated between 1 in 34,810 to 1 in 8,664 live male births [1][8] Company Overview - Sentynl Therapeutics, a biopharmaceutical company focused on rare diseases, is responsible for the development and commercialization of CUTX-101 [4][9] - The company was acquired by Zydus Group in 2017 and aims to address unmet medical needs through innovative therapies [9][10] - Fortress Biotech, which previously advanced the CUTX-101 program, continues to support its development [5][11] Product Details - CUTX-101 is a subcutaneous injectable formulation of copper histidinate, designed to improve tolerability and enhance neurodevelopmental outcomes in Menkes disease patients [6][7] - The drug has received multiple FDA designations, including Breakthrough Therapy and Orphan Drug Designation, indicating its potential significance in treating this condition [3][6] Market Context - There are currently no FDA-approved treatments for Menkes disease, highlighting a significant unmet need in the pediatric healthcare market [8][12] - The ongoing clinical trials and positive results for CUTX-101 may position it as a critical therapeutic option for affected children and their families [2][5]