International Medical News The U.S. Food and Drug Administration (FDA) has officially granted approval to Zycubo (copper histidinate), making it the first sanctioned therapy in the United States for Menkes disease. This rare genetic neurodegenerative disorder previously had no approved pharmacological treatment.
Key Highlights:
-
Mechanism of Action: Zycubo is an injectable copper replacement therapy. It restores copper homeostasis and maintains essential mineral levels, bypassing the absorption impairments caused by genetic mutations.
-
Clinical Efficacy: Trials involving 66 patients demonstrated a dramatic increase in survival rates. The median overall survival for treated patients reached 177.1 months, compared to a mere 17.6 months in the untreated external control group.
-
Approval Timeline: The green light followed a swift turnaround after the manufacturer (Sentynl) resolved manufacturing compliance issues identified by the FDA in late 2025.
-
Disease Profile: Menkes disease primarily affects males (1 in 100,000 to 250,000 births). Without intervention, it leads to severe developmental delays and seizures, with a typical life expectancy of less than three years.
-
Commercial Value: The approval includes a rare pediatric disease priority review voucher (PRV). This voucher will be transferred to Cyprium Therapeutics (a Fortress Biotech subsidiary) as part of the development agreement with Sentynl (a Zydus Lifesciences subsidiary).