Zydus Lifesciences Gains USFDA Orphan Drug Designation for Desidustat

Zydus Lifesciences Ltd has announced that the United States Food and Drug Administration (USFDA) has granted Orphan Drug Designation to its drug Desidustat for the treatment of beta-thalassemia. This designation is intended to support the development of treatments for rare diseases affecting fewer than 200,000 people in the United States. The announcement was made on November 6, 2025, and highlights the potential benefits of Desidustat as a treatment option.

The Orphan Drug Designation provides Zydus with eligibility for certain development incentives. These incentives include tax credits for qualified clinical testing and prescription drug user fee exemptions. Additionally, the designation offers the potential for a seven-year marketing exclusivity period, subject to USFDA approval. This exclusivity period is intended to encourage the development of treatments for rare diseases by providing a competitive advantage in the market.

Desidustat is a hypoxia inducible factor-prolyl hydroxylase inhibitor (HIF-PHI). It has the potential to increase hemoglobin and red blood cell counts, which are critical factors in the treatment of beta-thalassemia. Research conducted on beta-thalassemic mice has shown that Desidustat treatment led to an increase in hemoglobin and red blood cell levels. This mechanism of action positions Desidustat as a promising candidate for addressing the medical needs of beta-thalassemia patients, who often require lifelong blood transfusions and treatment for iron overload.

The USFDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for rare diseases. Beta-thalassemia is characterized by low levels of hemoglobin, leading to a lack of oxygen in the body and resulting in symptoms such as weakness and fatigue. The condition often requires chronic blood transfusions, which can lead to complications such as iron overload. Desidustat’s potential to increase hemoglobin levels may offer a new therapeutic approach for managing this condition.

The announcement of the Orphan Drug Designation for Desidustat was made by Zydus on November 6, 2025. The company has not disclosed specific timelines for the further development or potential commercialization of Desidustat. The designation marks a significant step in the regulatory process, providing Zydus with the opportunity to advance the development of Desidustat for beta-thalassemia treatment.

Zydus Lifesciences Ltd is a global pharmaceutical company focused on discovering, developing, manufacturing, and marketing a broad range of healthcare therapies. The company employs over 29,000 people worldwide, including 1,500 scientists engaged in research and development. Zydus is committed to unlocking new possibilities in life sciences through quality healthcare solutions and has introduced several innovative products in the market over the last decade.