Zydus Receives USFDA Orphan Drug Designation for Desidustat in Beta-Thalassemia

Zydus Lifesciences has announced that the U.S. Food and Drug Administration (USFDA) has granted Orphan Drug Designation (ODD) to Desidustat, a novel oral HIF-prolyl hydroxylase inhibitor (HIF-PHI), for the treatment of beta-thalassemia. The designation supports the development of medicines for rare diseases affecting fewer than 200,000 people in the United States.

Advancing Treatment for Rare Blood Disorders

Beta-thalassemia is a genetic blood disorder characterized by reduced hemoglobin levels, causing fatigue, weakness, and severe complications that often require lifelong blood transfusions. Desidustat has shown the potential to increase hemoglobin and red blood cell counts by stimulating the body’s natural erythropoietic response. Preclinical research in beta-thalassemia models demonstrated improvements in hemoglobin and RBC levels following Desidustat treatment.

Regulatory Incentives and Future Development Path

The USFDA Orphan Drug Designation provides Zydus with several incentives, including tax credits for clinical testing, waiver of user fees, and seven years of market exclusivity upon approval. Dr. Sharvil Patel, Managing Director of Zydus Lifesciences, stated that the recognition highlights the urgent medical need to develop Desidustat for beta-thalassemia, reinforcing the company’s commitment to advancing therapies for rare and life-threatening conditions.