Copper Histidinate: Pediatric First Approval.

Copper histidinate (ZYCUBO®) is a bioavailable copper replacement therapy developed by Cyprium Therapeutics and Sentynl Therapeutics for the treatment of Menkes disease, a genetic disorder caused by variants in the copper transporter gene, ATP7A. Because copper cannot be absorbed from the gastrointestinal tract by individuals with Menkes disease, copper histidinate is administered as a daily subcutaneous injection. In January 2026, copper histidinate received its first approval in the USA by the U.S. Food and Drug Administration for the treatment of Menkes disease in pediatric patients. Orphan Designation was granted by The European Commission to copper histidinate for the treatment of Menkes disease. This article summarizes the milestones in the development of copper histidinate leading to this first approval for Menkes disease in pediatric patients.