copper histidine

Copper histidine is a subcutaneous injectable formulation of a copper-amino acid complex used as a therapeutic agent for Menkes disease, a rare and often fatal genetic disorder characterized by impaired copper transport and deficiency[1][3][5]. The drug acts as a heavy metal replacement therapy, delivering bioavailable copper directly into the bloodstream to compensate for the defective ATP7A transporter in Menkes disease patients[1][8]. Early treatment with copper histidine has been shown to improve neurodevelopmental outcomes and significantly increase survival rates compared to untreated controls[3][8]. The formulation known as CUTX-101 has received FDA Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designations. It is currently under regulatory review with an expected Prescription Drug User Fee Act (PDUFA) action date in 2025[3][5].

Brand names

Zycubo

Other names

copper histidinatecopper histidine subcutaneous