FDA Approves First Treatment for Children With Menkes Disease

The U.S. Food and Drug Administration today approved the Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients.
“With today’s action, children with this devastating, degenerative disease will have an FDA-approved treatment option and the potential to live longer,” said Christine Nguyen, M.D., Deputy Director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research. “The FDA will continue to work with the rare disease community to advance drug development for patients with Menkes disease and other rare conditions.”
Menkes disease is a neurodegenerative disorder caused by a genetic defect that impairs a child’s ability to absorb copper. The disease is characterized by seizures, failure to gain weight and grow, developmental delays, and intellectual disability. It leads to abnormalities of the vascular system, bladder, bowel, bones, muscles, and nervous system. Children with classical Menkes (90% of those with the disease) begin to develop symptoms in infancy and typically do not live past three years. It affects approximately one in every 100,000-250,000 live births worldwide and is more common in boys.