Why in news?
The U.S. Food and Drug Administration has approved Zycubo, a copper histidinate injection, as the first treatment for children with Menkes disease. The approval on 12 January 2026 followed promising clinical trial results showing that early administration of the drug can significantly improve survival in affected infants.
Background
Menkes disease is a rare genetic disorder caused by mutations in the ATP7A gene. This mutation impairs the body’s ability to transport copper, an essential element for the development of the brain, bones and connective tissues. As a result, copper accumulates in some tissues while being deficient in others. Symptoms typically appear in infancy and may include sparse or kinky hair, seizures, poor muscle tone, developmental delays and growth failure. Most children with untreated Menkes disease do not survive beyond three years. The condition is inherited in an X‑linked recessive pattern, so it primarily affects boys.
Zycubo and its benefits
- Copper replacement: Zycubo delivers copper directly into the bloodstream, bypassing the defective transport protein. This helps restore copper levels in tissues where it is needed.
- Clinical outcomes: In clinical studies involving 66 treated patients, early treatment before symptoms appeared reduced the risk of death or severe disability by about 78 percent compared with untreated children.
- Early intervention: The therapy is most effective when started within a few weeks of birth, highlighting the importance of newborn screening and genetic counselling.
- Side effects: Common adverse reactions include fever, vomiting and irritation at the injection site. Because Menkes disease is life‑threatening, the benefits of treatment outweigh these manageable risks.
- Regulatory recognition: The drug received Orphan Drug, Breakthrough Therapy, Fast‑Track and Priority Review designations from the FDA, underscoring its significance for a rare disease with no prior treatment options.
Significance and next steps
- This approval offers hope to families affected by Menkes disease, as early copper supplementation can markedly prolong life and improve neurological outcomes.
- Screening programmes for newborns will be crucial to identify affected infants before symptoms appear, allowing timely initiation of therapy.
- The development of Zycubo encourages further research into gene therapies and other interventions that might address the underlying genetic cause.
- Families should seek genetic counselling when there is a history of Menkes disease, as carriers can be identified and supported during pregnancy and after delivery.
