Zydus Lifesciences Unit Secures Landmark FDA Approval for Rare Disease Drug
Overview
Zydus Lifesciences' US subsidiary, Sentynl Therapeutics, has achieved a significant regulatory milestone with US FDA approval for ZYCUBO (copper histidinate), marking the first therapy for Menkes disease. This rare genetic disorder previously lacked any approved treatment in the U.S. Clinical data demonstrated an 80% reduction in mortality risk and vastly improved survival rates in pediatric patients, offering critical hope.
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Regulatory Milestone Achieved
Zydus Lifesciences Limited's U.S.-based subsidiary, Sentynl Therapeutics Inc., has secured approval from the U.S. Food and Drug Administration (USFDA) for ZYCUBO® (copper histidinate). This landmark decision designates ZYCUBO® as the first and only approved therapy for Menkes disease in the United States, addressing a critical unmet medical need. The approval was filed with exchanges on Tuesday, January 13.
The Disease and the Treatment
Menkes disease is a rare, often fatal X-linked recessive genetic disorder resulting from impaired copper transport. This deficiency leads to severe neurological damage and early mortality, with most untreated patients not surviving beyond two to three years of age. ZYCUBO® is a subcutaneous injectable formulation of copper histidinate, meticulously designed to restore copper balance and maintain necessary levels in affected pediatric patients.
Clinical Efficacy Data
The US drug regulator's approval is substantiated by compelling clinical data. Studies revealed an approximately 80% reduction in the risk of death for pediatric patients receiving early treatment with ZYCUBO®, compared to an untreated external control group. Median overall survival saw a dramatic improvement, extending to 177.1 months, a significant increase from the 17.6 months observed in untreated patients.
Strategic Significance and Expert Opinion
Dr. Sharvil P. Patel, Managing Director of Zydus Lifesciences, called the approval a transformative moment, offering hope where none existed. Matt Heck, CEO of Sentynl, emphasized the drug's safety and effectiveness. Dr. Stephen Kaler, a clinical genetics specialist, noted this as the culmination of decades of research, stressing the importance of early diagnosis. This approval reinforces Zydus Lifesciences' strategic focus on advancing innovative therapies for rare diseases globally.
