The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Desidustat, a novel oral Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitor (HIF-PHI), for the treatment of beta-thalassemia. The USFDA's Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States (US).

Speaking on the development, Dr Sharvil Patel, Managing Director, Zydus Lifesciences, said, “This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop Desidustat to address beta-thalassemia.”

Beta thalassaemia patients have low levels of haemoglobin, which results in a lack of oxygen in many parts of the body, leading to weakness, fatigue and more serious complications. Treatment for people with beta thalassaemia often requires lifelong regimens of chronic blood transfusions for survival and treatment for iron overload due to the transfusions.

Desidustat has the potential to increase haemoglobin and Red Blood Cell (RBC) counts. Research in beta-thalassaemic mice showed that Desidustat treatment led to an increase in haemoglobin and RBC levels. ODD by the USFDA for Desidustat provides eligibility for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and a potential seven-year marketing exclusivity upon the USFDA approval.