U.S. FDA Approves First Menkes Disease Treatment for Children, Offering New Option for Families

Washington, D.C. — Families facing Menkes disease—a rare, rapidly progressive disorder that disrupts how infants absorb and use copper—now have the first FDA-approved treatment aimed at improving survival.

On Monday, the U.S. Food and Drug Administration approved Zycubo (copper histidinate) injection for pediatric patients with Menkes disease. The therapy is designed to replace copper in a form delivered by subcutaneous injection, bypassing the intestinal absorption problem caused by the genetic defect, the agency said.

Menkes disease can cause seizures, failure to grow, developmental delays, and widespread effects on blood vessels, bones, muscles, and the nervous system. In “classical” cases—reported by the FDA as the majority—symptoms typically begin in infancy and many children do not live beyond early childhood without effective treatment.

The FDA said it evaluated Zycubo in two open-label, single-arm trials, comparing outcomes for 66 treated children to 17 untreated children from external control groups. Children who started treatment within four weeks of birth had a 78% lower risk of death than untreated children, and nearly half of those treated early lived beyond age 6; some lived beyond age 12.

The most common side effects included infections, respiratory problems, seizures, vomiting, fever, anemia, and injection-site reactions. Because copper can build up in the body, the FDA said children receiving Zycubo should be closely monitored for toxicity.

What parents should do: If Menkes disease is suspected or diagnosed, ask your child’s care team how quickly treatment should begin, what monitoring labs and follow-ups will be needed, and what symptoms should prompt urgent care. For updates, families can check FDA announcements and their child’s specialty clinic.

This article was produced by an education parenting today journalist with the assistance of AI. This is not legal advice. All content is reviewed for accuracy and fairness.