Zydus Lifesciences Ltd, a leading global discovery-based pharmaceutical company, announced that the US Food and Drug Administration (USFDA) has granted Orphan Drug Designation (ODD) to its novel oral drug Desidustat for the treatment of beta-thalassemia.
The Orphan Drug Designation is given by the USFDA’s Office of Orphan Products Development to support the development of medicines for rare diseases affecting fewer than 200,000 people in the United States.
Commenting on the development, Dr. Sharvil Patel, Managing Director of Zydus Lifesciences, said, “This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop Desidustat to address beta-thalassemia.”
Beta-thalassemia is a rare inherited blood disorder in which patients have abnormally low levels of haemoglobin, leading to insufficient oxygen supply in the body. This often results in weakness, fatigue, and severe complications. Patients typically require lifelong blood transfusions and treatment for iron overload caused by frequent transfusions.
Desidustat is a hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) that has shown potential to increase haemoglobin and red blood cell (RBC) levels. Preclinical studies in beta-thalassemia models have indicated that Desidustat treatment significantly improved haemoglobin and RBC counts.
