Zydus Lifesciences Limited has achieved a significant regulatory milestone with the FDA approval of ZYCUBO (copper histidinate) for treating Menkes disease in pediatric patients. The approval was announced through Sentynl Therapeutics Inc., a wholly-owned US subsidiary of the Indian pharmaceutical company, marking the first and only approved treatment for this rare and fatal genetic condition in the United States.

Breakthrough Treatment for Rare Disease

Menkes disease is a rare X-linked recessive pediatric condition caused by mutations in the ATP7A gene, which encodes a copper transporter. Patients are born unable to absorb dietary copper and have impaired copper transport across the blood-brain barrier. The disease affects an estimated 1 in 34,810 live male births, with potentially higher prevalence rates of 1 in 8,664 based on recent genome-based studies.

The condition presents with distinctive clinical features including sparse and depigmented "kinky hair," connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, failure to thrive, and neurodevelopmental delays. Most untreated patients do not survive beyond three years of age.

Clinical Efficacy and Safety Profile

ZYCUBO demonstrated compelling clinical results in pivotal studies, showing statistically significant improvement in overall survival for patients receiving early treatment. The therapy achieved a nearly 80% reduction in the risk of death compared to untreated patients.

The subcutaneous injectable formulation delivers elemental copper daily to restore copper homeostasis and maintain appropriate copper levels in affected patients. However, treatment requires careful monitoring due to potential copper accumulation and related toxicity, particularly affecting kidney, liver, and hematopoietic systems.

Common adverse reactions observed in clinical trials included pneumonia (30%), viral infection (27%), respiratory failure (23%), seizure (23%), bacterial infection (20%), hemorrhage (18%), and hypotension (16%). Healthcare providers must conduct periodic laboratory monitoring of kidney function, liver function, and complete blood counts during treatment.

Regulatory Recognition and Development Path

The FDA granted ZYCUBO multiple designations recognizing its importance for this underserved patient population:

• Breakthrough Therapy designation
• Fast Track designation
• Rare Pediatric Disease designation
• Orphan Drug designation

The European Medicines Agency has also granted Orphan Designation for copper histidinate. Sentynl acquired the therapy from Cyprium Therapeutics in 2023 and advanced it through final development stages.

Management Commentary and Future Impact

"Approval is a pivotal step towards achieving our goal of making a meaningful impact on patients, caregivers, and the rare disease community," stated Dr. Sharvil P. Patel, Managing Director of Zydus Lifesciences Limited. "This milestone marks a transformative moment for the Zydus Group and for families affected by Menkes disease."

Matt Heck, CEO of Sentynl, emphasized the significance for patient care: "FDA's approval serves as compelling affirmation that a safe and effective therapy is now available for patients living with this devastating disease."

About the Companies

Zydus Lifesciences Limited operates as an innovation-led life sciences company with global presence across the United States, India, and international markets. As of September 30, 2025, the group employs 29,000 people worldwide, including 1,500 scientists engaged in research and development activities.

Sentynl Therapeutics Inc. functions as a commercial-stage biopharmaceutical company focused on rare disease treatments, leveraging both its US operations and parent organization resources to advance therapy development and delivery to patients globally.

Zydus Lifesciences Limited has announced the dissolution of its French subsidiary Ampliman 2, effective January 9, 2026, as part of a corporate restructuring that will not affect the pharmaceutical company's business operations. The announcement was made through a regulatory filing under Regulation 30 of the Securities and Exchange Board of India (Listing Obligations and Disclosure Requirements) Regulations, 2015.

Subsidiary Structure and Dissolution Details

Ampliman 2, France, operated as a wholly owned subsidiary of Zydus MedTech (France) S.A.S., which itself is a step-down wholly owned subsidiary of Zydus Lifesciences Limited . The dissolution was executed without liquidation, ensuring a smooth transfer of corporate assets and obligations.

Asset and Liability Transfer

Upon dissolution, all assets and liabilities of Ampliman 2 have been transferred to Zydus MedTech (France) S.A.S., ensuring continuity of operations and maintaining the company's European business structure. This transfer mechanism allows for the consolidation of resources while preserving operational capabilities within the French market.

Business Impact Assessment

The company has explicitly stated that the dissolution of Ampliman 2 will not impact its business operations. This indicates that the subsidiary's functions have been effectively integrated into the parent French entity or were non-critical to the overall business strategy. The restructuring appears to be aimed at simplifying the corporate structure while maintaining operational efficiency.

Regulatory Compliance

The dissolution announcement was signed by Dhaval N. Soni, Company Secretary and Compliance Officer, ensuring proper regulatory disclosure. The company maintains its registered office at Zydus Corporate Park in Ahmedabad, Gujarat, and continues to operate under CIN L24230GJ1995PLC025878. This corporate action demonstrates the company's commitment to transparent communication with stakeholders regarding structural changes in its international operations.