Zydus US Arm Gains FDA Approval For ZYCUBO In Pediatric Menkes Patients

Zydus Lifesciences announced that its U.S.-based biopharmaceutical subsidiary, Sentynl Therapeutics Inc., has received approval from the U.S. Food and Drug Administration (FDA) for ZYCUBO (copper histidinate) to treat Menkes disease in pediatric patients. This marks the first and only FDA-approved therapy for this rare and fatal genetic disorder in the United States.

Menkes disease is an X-linked recessive condition caused by mutations in the ATP7A gene, which impairs the body’s ability to absorb dietary copper and disrupts copper transport across the blood-brain barrier. Untreated patients typically do not survive beyond three years of age.

ZYCUBO, administered as a subcutaneous injectable, restores copper homeostasis and maintains copper levels in patients. Clinical trials demonstrated a statistically significant improvement in overall survival, with early-treated patients achieving a 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 observed in ≥7% of patients included pneumonia, viral and bacterial infections, respiratory failure, seizures, hypotension, vomiting, tachycardia, fever, volume depletion, fractures, dyspnea, elevated liver enzymes, diarrhea, fungal infections, anemia, and local injection-site reactions.

The FDA approval was described by Dr. Sharvil P. Patel, Managing Director of Zydus Lifesciences, as a transformative moment for the company and for families affected by Menkes disease, giving patients access to an approved therapy for the first time and bringing hope where no options previously existed.

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