Zydus Lifesciences has received final approval from the United States Food and Drug Administration (USFDA) for Eltrombopag Tablets in multiple strengths, marking another significant regulatory milestone for the pharmaceutical company. The company announced this development on Thursday, January 15, highlighting the approval's coverage of four different dosage strengths for thrombocytopenia treatment.

Product Specifications and Market Opportunity

The USFDA approval encompasses Eltrombopag Tablets in four distinct strengths, positioning the company to serve diverse patient needs in the thrombocytopenia treatment market. The approval represents access to a substantial market opportunity based on latest sales data.

Therapeutic Applications and Mechanism

Eltrombopag tablets are specifically indicated for treating thrombocytopenia, a condition characterized by low platelet count associated with certain blood disorders. The medication functions by stimulating bone marrow cells to increase platelet production, thereby helping lower bleeding risks for patients suffering from this condition.

The therapeutic approach addresses a critical medical need, as thrombocytopenia can lead to serious bleeding complications. By targeting the bone marrow's platelet production mechanism, Eltrombopag provides healthcare providers with an effective treatment option for managing this challenging condition.

Manufacturing and Production Details

Zydus Lifesciences will produce the approved Eltrombopag tablets at its formulation manufacturing facility located in the Special Economic Zone (SEZ) in Ahmedabad. This facility represents part of the company's established manufacturing infrastructure, ensuring quality production capabilities for the US market supply.

Regulatory Portfolio Expansion

The Eltrombopag approval contributes to Zydus Lifesciences' expanding regulatory portfolio in the United States pharmaceutical market. The latest approval brings the company's cumulative achievements to significant milestones in its regulatory journey.

The company has maintained an active filing strategy since commencing its ANDA submission process, resulting in a substantial portfolio of approved products. This latest approval reinforces the company's commitment to expanding its presence in the competitive US generics market through strategic product development and regulatory execution.

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.