Sentynl Therapeutics Receives FDA Approval for ZYCUBO, First US Treatment for Rare Menkes Disease
Sentynl Therapeutics, a subsidiary of Zydus Lifesciences, received US FDA approval for ZYCUBO (copper histidinate) on January 13, 2026, as the first treatment for pediatric Menkes disease, a rare genetic disorder impairing copper absorption.
Menkes disease is an X-linked recessive disorder caused by ATP7A gene mutations, affecting about 1 in 8,664 male births, with untreated patients typically dying by age three.
Clinical trials showed early treatment (within 4 weeks of birth) reduced death risk by nearly 80%, with median survival of 177 months versus 17.6 months in untreated patients.
ZYCUBO is a subcutaneous injection that bypasses impaired intestinal copper absorption; common side effects include pneumonia, seizures, and respiratory distress.
The approval followed a resubmission after a September 2025 complete response letter on manufacturing issues; it received Breakthrough Therapy, Fast Track, and Orphan Drug designations.