Zydus Receives FDA Orphan Drug Designation for Desidustat in Beta-Thalassemia Treatment

Key Insights

• Zydus Lifesciences has received FDA Orphan Drug Designation for Desidustat, a novel oral HIF-PHI compound, for treating beta-thalassemia.
• The designation supports development of medicines for rare diseases affecting fewer than 200,000 people in the United States.
• Desidustat works as a hypoxia inducible factor-prolyl hydroxylase inhibitor with potential to increase hemoglobin and red blood cell counts.

Ahmedabad-based Zydus Lifesciences has received Orphan Drug Designation from the U.S. Food and Drug Administration for Desidustat, a novel oral hypoxia inducible factor-prolyl hydroxylase inhibitor (HIF-PHI), for the treatment of beta-thalassemia. The designation underscores the urgent medical need for new therapeutic options in this rare blood disorder.

Addressing Unmet Medical Need in Beta-Thalassemia

Beta-thalassemia patients experience low hemoglobin levels, resulting in insufficient oxygen delivery throughout the body and leading to weakness, fatigue, and serious complications. Current treatment paradigms often require lifelong chronic blood transfusions for survival, accompanied by iron overload management due to repeated transfusions.

"This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop Desidustat to address beta-thalassemia," said Dr. Sharvil Patel, Managing Director of Zydus Lifesciences Limited.

Novel Mechanism of Action Shows Promise

Desidustat functions as a hypoxia inducible factor-prolyl hydroxylase inhibitor, offering potential to increase hemoglobin and red blood cell counts through a distinct therapeutic pathway. Preclinical research conducted in beta-thalassemic mice demonstrated that desidustat treatment led to increased hemoglobin and red blood cell levels, providing early evidence of the compound's therapeutic potential.

Regulatory Benefits and Development Incentives

The FDA's Office of Orphan Drug Products grants orphan status to support development of medicines for rare diseases affecting fewer than 200,000 people in the United States. This designation provides Zydus with several development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.

The orphan drug designation represents a significant milestone for Zydus as the company advances this novel therapeutic approach for beta-thalassemia patients who currently face limited treatment options beyond chronic transfusion therapy.