Zydus Lifesciences-owned Sentynl Therapeutics has received United States Food and Drug Administration approval for Zycubo, also known as copper histidinate, for the treatment of Menkes disease in paediatric patients. This marks the first and only approved therapy for this rare and fatal genetic disorder in the United States.
What is Menkes Disease?
Menkes disease is a neurodegenerative disorder caused by a genetic defect that prevents the body from properly absorbing copper. It mainly affects infants and young children. The condition can lead to seizures, poor weight gain, growth failure, developmental delays, and intellectual disability. It also affects the vascular system, bladder, bowel, bones, muscles, and nervous system.
About Zycubo
Zycubo is a copper replacement therapy administered through subcutaneous injection. The drug was acquired by Sentynl Therapeutics from Cyprium Therapeutics in 2023. It was then advanced through late-stage development and supported by positive very important study results. The product had earlier received breakthrough therapy, fast track, rare paediatric disease, and orphan drug designations from the US FDA.
Significance of the Approval
The approval is important because Menkes disease had no approved treatment option in the US before this decision. The availability of an approved therapy offers a new clinical option for affected children and their families. Copper histidinate has also received orphan designation from the European Medicines Agency, indicating recognition of its potential in treating a rare disease.
Regulatory and Global Context
The US FDA approval reflects growing regulatory support for therapies targeting rare paediatric disorders. Orphan and rare disease designations are designed to encourage drug development for conditions with limited treatment options. The approval may also strengthen interest in similar rare disease therapies in other markets.
Last Modified: April 26, 2026