This marks the first and only FDA-approved therapy for this rare and fatal genetic disorder in the United States. ZYCUBO' is not indicated for Occipital Horn Syndrome.
Menkes disease, an X-linked recessive pediatric condition caused by mutations in the ATP7A gene, prevents patients from absorbing dietary copper, resulting in impaired copper transport across the blood-brain barrier. Untreated patients typically do not survive beyond three years of age.
ZYCUBO is a subcutaneous injectable formulation that restores copper homeostasis and maintains copper levels in patients. Clinical trials demonstrated a statistically significant improvement in overall survival, with the early treatment cohort showing median survival of 177.1 months compared to 17.6 months for untreated patients, representing a nearly 80% reduction in risk of death. Common adverse reactions (≥7% incidence) included pneumonia, viral infection, respiratory failure, seizure, bacterial infection, hemorrhage, hypotension, vomiting, tachycardia, pyrexia, volume depletion, fracture, dyspnea, elevated transaminases, diarrhea, fungal infection, anemia, and local administration reactions.
Commenting on the approval, Dr. Sharvil P. Patel, MD, Zydus Lifesciences, said, '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.'
Matt Heck, CEO of Sentynl, added, 'FDA's approval serves as compelling affirmation that a safe and effective therapy is now available for patients living with this devastating disease.'
Dr. Stephen Kaler, clinical genetics specialist at Columbia University Medical Center, said, 'Beginning copper histidinate therapy in affected neonates has been shown to reduce symptoms and prolong life. Increased awareness and rapid testing are critical.'
Sentynl, which acquired ZYCUBO' from Cyprium Therapeutics in 2023, advanced the drug through final FDA review, receiving Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations. Copper histidinate has also been granted Orphan Drug status by the European Medicines Agency.
This approval is expected to bring new treatment options to the rare disease community, reinforcing Zydus Group's presence in the global pediatric therapeutics space.
The Zydus Group, with an overarching purpose of empowering people with freedom to live healthier and more fulfilled lives, is an innovative, global life-sciences company that discovers, develops, manufactures, and markets a broad range of healthcare therapies. The group employs over 27000 people worldwide and is driven by its mission to unlock new possibilities in life sciences through quality healthcare solutions that impact lives.
Zydus Lifesciences reported a 38.12% jump in consolidated net profit to Rs 1,258.60 crore in Q2 FY26, compared with Rs 911.2 crore posted in Q2 FY25. Revenue from operations increased 18.07% year on year (YoY) to Rs 6,037.9 crore in Q2 FY26.
Shares of Zydus Lifesciences shed 0.15% to Rs 895 on the BSE.
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