Sentynl Therapeutics Inc. (Sentynl), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences (Zydus Group), announced that the U.S. Food and Drug Administration has approved ZYCUBO® (copper histidinate) for the treatment of Menkes disease in pediatric patients. This is the first and only treatment approved for Menkes disease, a rare and fatal genetic condition, in the United States. ZYCUBO® is not indicated for the treatment of Occipital Horn Syndrome.
Menkes disease is a rare X-linked recessive pediatric disease caused by mutations of the copper transporter ATP7A encoded by the ATP7A gene. Patients with Menkes disease are born with the inability to absorb dietary copper and subsequently have impaired copper transport across the blood-brain barrier, and, until now, there has been no approved treatment in the United States. ZYCUBO® is a subcutaneous injectable formulation of copper histidinate that restores copper homeostasis and maintains copper levels in patients with Menkes disease.