Sentynl Therapeutics Inc, a US-based biopharmaceutical company wholly owned by Zydus Lifesciences Limited, has received approval from the US Food and Drug Administration (FDA) for ZYCUBO® (copper histidinate) for the treatment of pediatric patients with Menkes disease. ZYCUBO® is the first and only FDA-approved therapy for this rare and life-threatening genetic disorder in the United States. The drug is not approved for the treatment of Occipital Horn Syndrome.
Menkes disease is a rare X-linked recessive disorder caused by mutations in the ATP7A gene, leading to impaired copper absorption and transport, particularly to the brain. Until now, patients in the US had no approved treatment options. ZYCUBO® is a subcutaneous injectable formulation of copper histidinate designed to restore copper balance and maintain essential copper levels in affected patients.
Dr Sharvil P Patel, Managing Director, Zydus Lifesciences Limited, said the approval represents a defining moment for families affected by Menkes disease, offering access to a proven therapy for the first time and reinforcing the group’s commitment to addressing unmet medical needs in rare diseases. Matt Heck, Chief Executive Officer of Sentynl, said the FDA decision confirms the availability of a safe and effective treatment for a condition where most untreated children do not survive beyond early childhood.
The approval is supported by positive clinical outcomes showing a significant improvement in overall survival among patients who received early treatment with ZYCUBO®. Data demonstrated nearly an 80 percent reduction in the risk of death compared to untreated patients, with median overall survival extending to 177.1 months versus 17.6 months in the external control cohort.
Dr Stephen Kaler of Columbia University Medical Center highlighted the importance of early diagnosis and prompt initiation of copper histidinate therapy, noting its potential to reduce disease severity and extend life when started in newborns.
Sentynl acquired ZYCUBO® from Cyprium Therapeutics in 2023 and advanced the therapy through late-stage development with FDA support, including Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations. Copper histidinate has also received Orphan Drug designation from the European Medicines Agency.
Zydus Lifesciences Limited is a global, innovation-driven life sciences company with operations across pharmaceuticals, consumer wellness, and emerging MedTech segments. Sentynl Therapeutics focuses on delivering treatments for rare diseases, leveraging the global capabilities of the Zydus Group to expand patient access worldwide.
