Zydus entity Sentynl’s drug for Menkes disease gets U.S. FDA nod

U.S. biopharmaceutical company Sentynl Therapeutics Inc, which is wholly owned by Zydus Lifesciences, has received the U.S. Food and Drug Administration approval for Zycubo (copper histidinate) for treatment of Menkes disease in paediatric patients.

This is the first and only treatment approved for Menkes disease, which is a rare and fatal genetic condition, in the U.S., Zydus said on Tuesday.

Sentynl had acquired Zycubo from Cyprium Therapeutics in 2023 and advanced the product through the final stages of development with the U.S. FDA based on positive results from pivotal studies, receiving breakthrough therapy, fast track, rare paediatric disease and the FDA orphan drug designations.

For the first time, patients have access to an approved therapy, offering hope where no options existed, Zydus Lifesciences managing director Sharvil P. Patel said in a release.

Copper histidinate has also been granted orphan designation by the European Medicines Agency.

Separately, in a release announcing the approval, the U.S. FDA said 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. Zycubo is a copper replacement therapy given by subcutaneous injection.