Ahmedabad: Zydus Lifesciences US based subsidiary, Sentynl Therapeutics, has received US FDA’s approval for its ZYCUBO® drug aimed at treating the rare genetic Menkes disease that affects children. This is the first and only treatment in the US for this disease, which generally affects just 1 in about 8,664 live births, mostly male. Worldwide, it is estimated that just 1 in about 250,000 live births.
Menkes disease is caused by mutations of the copper transporter ATP7A encoded in the ATP7A gene. Patients born with this disease are unable to absorb dietary copper and have impaired copper transport due ot it. Until now, there was no approved treatment for this disorder in the US.
ZYCUBO® aims to address this through its copper histidinate solution. Based on clinical trials, ZYCUBO® reduces the risk of death by almost 80%.
“Menkes disease presents significant challenges for patients and their families. With no known cure, most untreated patients do not survive beyond three years of age,” Matt Heck, CEO, Sentynl, said in a press release.
“FDA’s approval serves as compelling affirmation that a safe and effective therapy is now available for patients living with this devastating disease.” he added.
“Approval is a pivotal step towards achieving our goal of making a meaningful impact on patients, caregivers, and the rare disease community,” Dr. Sharvil P. Patel, Managing Director, Zydus Lifesciences Limited, added.
“This milestone marks a transformative moment for the Zydus Group and for families affected by Menkes disease. For the first time, patients have access to an approved therapy, offering hope where no options existed. We are proud to advance patient care and deliver innovative solutions to those who need them most.”
