USFDA6 Nov 2025, 10:00 am
Zydus Lifesciences Receives USFDA Orphan Drug Designation for Desidustat in Beta-Thalassemia Treatment
AI Summary
Zydus Lifesciences Ltd. has received Orphan Drug Designation (ODD) from the USFDA for Desidustat, a novel oral HIF-PHI, for the treatment of beta-thalassemia. Desidustat has the potential to increase haemoglobin and red blood cell counts. This designation 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 USFDA approval.
Key Highlights
- Zydus Lifesciences Ltd. receives Orphan Drug Designation (ODD) from the USFDA for Desidustat in the treatment of beta-thalassemia.
- Desidustat is a novel oral HIF-PHI with the potential to increase haemoglobin and red blood cell counts.
- Orphan drug designation 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 USFDA approval.
- Beta thalassemia patients have low levels of haemoglobin, leading to a lack of oxygen in many parts of the body, requiring lifelong regimens of chronic blood transfusions for survival.
- Desidustat research in beta-thalassaemic mice showed an increase in haemoglobin and red blood cell (RBC) levels.