Zydus Lifesciences announced that Sentynl Therapeutics Inc., a wholly-owned subsidiary, has received FDA approval for ZYCUBO® (copper histidinate) for treating Menkes disease in pediatric patients. This marks the first and only approved therapy for this rare and fatal genetic condition in the United States. The approval is based on clinical data demonstrating significant survival improvements with early treatment, offering new hope for affected families.
FDA Approves ZYCUBO for Menkes Disease
Sentynl Therapeutics Inc., a subsidiary of Zydus Lifesciences, has received U.S. FDA approval for ZYCUBO® (copper histidinate). This drug is indicated for the treatment of Menkes disease in pediatric patients. The announcement was made on January 13, 2026.
Significance of the Approval
ZYCUBO® is now the first and only therapy approved for treating Menkes disease in the United States. Menkes disease is a rare, fatal genetic condition.
Supporting Data
The FDA approval is supported by positive clinical trial results, which demonstrated a statistically significant improvement in overall survival for Menkes disease patients who received early treatment with ZYCUBO. The data showed a nearly 80% reduction in the risk of death compared to an untreated control group.
Survival Rates
Median overall survival for the ZYCUBO early treatment group was 177.1 months, compared to 17.6 months for the untreated control group.
About Menkes Disease
Menkes disease is a rare X-linked recessive pediatric disease caused by mutations of the copper transporter ATP7A. Patients are born with the inability to absorb dietary copper, leading to impaired copper transport. Without treatment, many patients do not survive beyond three years of age.
Adverse Reactions
The most common adverse reactions (incidence ≥7%) included pneumonia (30%), viral infection (27%), respiratory failure (23%), seizure (23%), bacterial infection (20%), hemorrhage (18%), and hypotension (16%).
Sentynl’s Commitment
Sentynl Therapeutics is dedicated to bringing innovative therapies to patients living with rare diseases, focusing on improving patient outcomes and access.
Source: BSE
