Zydus Lifesciences' unit Sentynl wins USFDA nod for rare pediatric disease therapy

Zydus

• ZYCUBO gets USFDA approval as first Menkes disease treatment in the US
• Early ZYCUBO treatment cuts death risk by 80 percent in clinical trials
• Zydus Lifesciences enters rare disease space with breakthrough Menkes therapy

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Sentynl Therapeutics, a U.S.-based subsidiary of Zydus Lifesciences, has secured USFDA approval for ZYCUBO (copper histidinate), the first and only treatment for Menkes disease in the United States.

The rare, fatal genetic disorder affects copper absorption and transport, leaving patients with severe neurological symptoms and a life expectancy of less than three years without treatment.

The approval marks a milestone for Zydus Lifesciences, positioning the group in the rare disease space with a therapy that addresses a critical unmet need.

“For the first time, patients have access to an approved therapy, offering hope where no options existed,” said Dr. Sharvil Patel, Managing Director of Zydus Lifesciences.

ZYCUBO, a subcutaneous injectable formulation, restores copper homeostasis in pediatric patients.

Clinical trials showed early treatment reduced the risk of death by nearly 80 percent compared to untreated controls, with median overall survival reaching 177 months versus 17.6 months for untreated patients.

The USFDA granted ZYCUBO multiple designations, including breakthrough therapy, Fast Track, and Orphan Drug status.

Sentynl acquired the asset from Cyprium Therapeutics in 2023 and advanced it through pivotal studies. The therapy also holds Orphan Designation in Europe.

What is Menkes disease?

Menkes disease, an X-linked recessive disorder caused by ATP7A gene mutations, has an estimated birth prevalence as high as 1 in 8,664 live male births. Untreated patients typically die by age three. ZYCUBO is not indicated for Occipital Horn Syndrome.

The approval underscores Zydus Lifesciences’ push into innovation-led therapies beyond its generics base. The company employs 29,000 globally and aims to expand its rare disease portfolio through Sentynl’s U.S. platform.