The U.S. Food and Drug Administration (FDA) has approved the
Zycubo (
copper histidinate) injection as the first treatment for
Menkes disease in pediatric patients.
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"With (the) action, children with this devastating, degenerative disease will have and 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 development for patients with Menkes disease and other rare conditions."
According to FDA, Menkes disease is a
neurodegenerative disorder caused by a genetic defect that impairs a child's ability to absorb copper. The disease is characterised 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 percent 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 is more common in boys, said FDA.
Zycubo is a
copper replacement therapy given by subcutaneous injection. It delivers copper in a form that bypasses the genetic defect in intestinal absorption, allowing the body to better use the mineral, said FDA.
The FDA evaluated Zycubo in two
open-label, single-arm clinical trials in pediatric patients treated for up to three years. Overall survival was assessed by comparing treated patients to untreated patients from contemporaneous external control groups. The analysis included 66 treated patients and 17 untreated patients, most of whom were from the United States, said FDA.
Children who began treatment within four weeks of birth had a 78 percent reduction in the risk of death compared with untreated patients. Nearly half of early-treated patients survived beyond six years, and some survived more than 12 years. No patients in the untreated control group survived beyond six years. Children who started treatment later than four weeks after birth also experienced a substantial benefit, according to FDA.
Side Effects
FDA said that the most common side effects reported for Zycubo included infections, respiratory problems, seizures, vomiting, fever, anemia and injection site reactions. Because copper can accumulate in the body, patients receiving Zycubo should be closely monitored for potential toxicity.
"This approval marks an unprecedented advance for children with Menkes disease,"
said Tracy Beth Hoeg, M.D., Ph.D.,
Acting Director of CBER. "The company demonstrated a large improvement in overall survival compared with untreated patients, using an innovative trial design that addressed the challenges of studying an ultra-rare disease."
This blog is for informational purposes only. Consult your doctor for proper diagnosis and medical treatment.
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