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Zydus Lifesciences Limited's US-based subsidiary Sentynl Therapeutics Inc. has received approval from the US Food and Drug Administration (USFDA) for ZYCUBO® (copper histidinate), making it the first and only approved therapy for Menkes disease in the United States.
In a press release filed with the exchanges on Tuesday, January 13, the company said ZYCUBO® has been approved for the treatment of pediatric patients with Menkes disease, a rare and often fatal genetic disorder caused by impaired copper transport in the body. Until now, there had been no FDA-approved treatment available for the condition in the US.
Menkes disease is an X-linked recessive disorder resulting from mutations in the ATP7A gene, which prevents proper absorption and transport of dietary copper, leading to severe neurological damage and early mortality if untreated. Most untreated patients do not survive beyond two to three years of age, the company noted.
Dr. Sharvil P. Patel, Managing Director of Zydus Lifesciences said, “This milestone marks a transformative moment for families affected by Menkes disease. For the first time, patients have access to an approved
therapy, offering hope where no options existed."
ZYCUBO® is a subcutaneous injectable formulation of copper histidinate designed to restore copper balance and maintain copper levels in affected patients.
Read more: Zydus Life's US arm gets FDA nod for paediatric drug CUTX-101 NDA resubmission
Matt Heck, CEO, Sentynl said, “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.” However, he added, “FDA’s approval serves as compelling affirmation that a safe and effective therapy is now available for patients living with this devastating disease.”
According to Zydus Life, the US drug regulator approval is backed by positive clinical data showing a nearly 80% reduction in the risk of death in patients who received early treatment with ZYCUBO®, compared with an untreated external control group. Median overall survival improved sharply to 177.1 months, versus 17.6 months in untreated patients, the pharma major said.
Also Read: Zydus Life unit partners with Formycon to supply immunotherapy drug Keytruda biosimilar in US, Canada
Calling the approval “the culmination of decades of research” into Menkes disease, Dr. Stephen Kaler, a clinical genetics and genomics specialist at Columbia University Medical Center, said increased awareness of the disease and early diagnosis is crucial, noting that starting copper histidinate therapy in affected newborns has been shown to reduce symptoms and prolong life.
ZYCUBO® was acquired by Sentynl from Cyprium Therapeutics in 2023 and has received multiple regulatory designations, including Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug status from the FDA. Copper histidinate has also been granted orphan designation by the European Medicines Agency.
Zydus Lifesciences said the approval reinforces its growing presence in the rare disease space and aligns with its focus on advancing innovative therapies for unmet medical needs globally.
Shares of the company were trading 894.80 as of 10.57 am. The stock has fallen 7.60% in the last six months.
In a press release filed with the exchanges on Tuesday, January 13, the company said ZYCUBO® has been approved for the treatment of pediatric patients with Menkes disease, a rare and often fatal genetic disorder caused by impaired copper transport in the body. Until now, there had been no FDA-approved treatment available for the condition in the US.
Menkes disease is an X-linked recessive disorder resulting from mutations in the ATP7A gene, which prevents proper absorption and transport of dietary copper, leading to severe neurological damage and early mortality if untreated. Most untreated patients do not survive beyond two to three years of age, the company noted.
Dr. Sharvil P. Patel, Managing Director of Zydus Lifesciences said, “This milestone marks a transformative moment for families affected by Menkes disease. For the first time, patients have access to an approved
therapy, offering hope where no options existed."
ZYCUBO® is a subcutaneous injectable formulation of copper histidinate designed to restore copper balance and maintain copper levels in affected patients.
Read more: Zydus Life's US arm gets FDA nod for paediatric drug CUTX-101 NDA resubmission
Matt Heck, CEO, Sentynl said, “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.” However, he added, “FDA’s approval serves as compelling affirmation that a safe and effective therapy is now available for patients living with this devastating disease.”
According to Zydus Life, the US drug regulator approval is backed by positive clinical data showing a nearly 80% reduction in the risk of death in patients who received early treatment with ZYCUBO®, compared with an untreated external control group. Median overall survival improved sharply to 177.1 months, versus 17.6 months in untreated patients, the pharma major said.
Also Read: Zydus Life unit partners with Formycon to supply immunotherapy drug Keytruda biosimilar in US, Canada
Calling the approval “the culmination of decades of research” into Menkes disease, Dr. Stephen Kaler, a clinical genetics and genomics specialist at Columbia University Medical Center, said increased awareness of the disease and early diagnosis is crucial, noting that starting copper histidinate therapy in affected newborns has been shown to reduce symptoms and prolong life.
ZYCUBO® was acquired by Sentynl from Cyprium Therapeutics in 2023 and has received multiple regulatory designations, including Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug status from the FDA. Copper histidinate has also been granted orphan designation by the European Medicines Agency.
Zydus Lifesciences said the approval reinforces its growing presence in the rare disease space and aligns with its focus on advancing innovative therapies for unmet medical needs globally.
Shares of the company were trading 894.80 as of 10.57 am. The stock has fallen 7.60% in the last six months.
