The FDA on Jan. 12 approved Zycubo injection as the first treatment for children with Menkes disease.
Menkes disease is a rare neurodegenerative disorder that hinders a child’s ability to absorb copper and can lead to seizures, growth and developmental delays, and intellectual disabilities. The average lifespan of a child with Menkes disease is about three years.
Zycubo is a copper replacement therapy developed by Sentynl Therapeutics. In clinical trials, patients who received treatment within a month of birth had a 78% lower mortality risk compared with untreated patients. In addition, nearly half of treated patients survived beyond six years.
“This approval marks an unprecedented advance for children with Menkes disease,” Tracy Beth Hoeg, MD, PhD, acting director of the FDA’s Center for Drug Evaluation and Research, said in a news release. “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.”
Zycubo received priority review, fast track designation, breakthrough therapy designation and orphan drug designation from the FDA.
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