Zydus Gets USFDA Orphan Drug Status for Beta-Thalassemia

New Delhi, Nov 6 (PTI) Zydus Lifesciences on Thursday said the US health regulator has granted Orphan Drug Designation (ODD) to Desidustat, a novel product for the treatment of beta-thalassemia.

The US Food and Drug Administration (USFDA) grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 2 lakh people in the US.

"This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop Desidustat to address beta-thalassemia," Zydus Lifesciences MD Sharvil Patel said in a regulatory filing.

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 is a hypoxia inducible factor (HIF)-prolyl hydroxylase inhibitor (PHI) and has the potential to increase haemoglobin and red blood cell counts.

This ODD 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.

Zydus Lifesciences shares were trading 0.71 per cent down at Rs 965.95 apiece on BSE.