Zydus Lifesciences Limited announced that its wholly-owned U.S. subsidiary, Sentynl Therapeutics Inc., has received approval from the U.S. Food and Drug Administration (FDA) for ZYCUBO® (copper histidinate). This marks the first and only approved therapy for Menkes disease, a rare and fatal genetic condition, in pediatric patients in the United States. ZYCUBO® is a subcutaneous injectable formulation of copper histidinate designed to restore copper homeostasis. The approval was supported by clinical efficacy results demonstrating a statistically significant improvement in overall survival for patients receiving early treatment with ZYCUBO®, showing a nearly 80% reduction in the risk of death compared to an untreated external control cohort. The median overall survival for the ZYCUBO® early treatment cohort was 177.1 months, compared to 17.6 months for the untreated cohort. Dr. Sharvil P. Patel, Managing Director of Zydus Lifesciences Limited, highlighted this as a pivotal step towards making a meaningful impact on patients and caregivers in the rare disease community. Menkes disease, an X-linked recessive pediatric disease, previously had no approved treatment in the United States, with most untreated patients not surviving beyond three years of age. Sentynl acquired the therapy from Cyprium Therapeutics in 2023 and has advanced its development, securing designations such as Breakthrough Therapy and Orphan Drug. The company also noted common adverse reactions including pneumonia, viral infection, and respiratory failure, and provided detailed safety information for healthcare providers.