Biopharmaceutical Company-Sentynl Therapeutics Inc. Announces FDA Approval of ZYCUBO
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This marks a historic milestone as the first and only treatment approved in the United States for Menkes disease, a rare and often fatal X-linked recessive genetic disorder.
Sentynl Therapeutics Inc., a subsidiary of Zydus Lifesciences Limited, has officially announced the FDA approval of ZYCUBO® (copper histidinate). This marks a historic milestone as the first and only treatment approved in the United States for Menkes disease, a rare and often fatal X-linked recessive genetic disorder. By providing a subcutaneous injectable formulation of copper histidinate, ZYCUBO helps restore copper homeostasis in pediatric patients who are otherwise unable to absorb the essential mineral due to mutations in the $ATP7A$ gene.
The approval is backed by compelling clinical data demonstrating that early intervention with ZYCUBO significantly alters the natural history of the disease. In pivotal studies, patients receiving early treatment showed a nearly 80% reduction in the risk of death compared to untreated historical groups. Most notably, the median overall survival for the treated cohort reached 177.1 months, a staggering improvement over the 17.6 months observed in the untreated control group, offering families a much-needed lifeline against the rapid progression of the condition.
Menkes disease typically manifests with distinct clinical features such as sparse, "kinky" hair, connective tissue issues, and severe neurological delays, with many untreated infants not surviving past three years of age. While ZYCUBO addresses this critical unmet need, the FDA notes that it is not indicated for Occipital Horn Syndrome. The treatment requires careful monitoring by healthcare providers, as the administration of copper can lead to accumulation and potential toxicity in the kidneys, liver, and hematopoietic system, particularly in very young infants with immature organ function.
Commonly reported adverse reactions include pneumonia, respiratory failure, and local administration site reactions. Despite these risks, the breakthrough status and subsequent approval of ZYCUBO represent a major advancement in rare disease therapeutics. Sentynl Therapeutics, which acquired the program from Cyprium Therapeutics in 2023, has successfully navigated the drug through Fast Track and Orphan Drug designations to bring this essential therapy to the pediatric population in the United States.
About Zydus Lifesciences Limited
Zydus Lifesciences Limited is an innovation-led life-sciences company with leadership positions across pharmaceuticals and consumer wellness, supported by an emerging MedTech franchise and a global footprint across the United States, India and other international markets. As of September 30, 2025, the group employs 29,000 people worldwide, including 1,500 scientists engaged in R&D, and is driven by its mission to unlock new possibilities in life sciences through quality healthcare solutions that impact lives. The group aspires to transform lives through path-breaking discoveries.
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