Zydus Lifesciences has received Orphan Drug Designation (ODD) from the USFDA for Desidustat, aimed at treating beta-thalassemia. Desidustat, an oral HIF-PHI, seeks to improve haemoglobin levels in patients. The ODD status provides Zydus with development incentives, including tax credits and potential marketing exclusivity upon USFDA approval. This designation underscores the need for therapies addressing this rare blood disorder, potentially affecting fewer than 200,000 people in the US.
Orphan Drug Status for Beta-Thalassemia Treatment
Zydus Lifesciences has announced that the USFDA has granted Orphan Drug Designation (ODD) to Desidustat, a novel oral HIF-PHI, for the treatment of beta-thalassemia. The announcement was made on November 6, 2025.
Desidustat: Addressing a Critical Need
Desidustat is designed as a hypoxia inducible factor (HIF)-prolyl hydroxylase inhibitor (PHI) with the potential to increase haemoglobin and red blood cell counts in beta-thalassemia patients. These patients often experience low haemoglobin levels, leading to oxygen deficiency and related complications.
Benefits of Orphan Drug Designation
The Orphan Drug Designation provides Zydus with certain development incentives. These include tax credits for qualified clinical testing and potential exemptions from prescription drug user fees. Moreover, upon USFDA approval, Zydus may be eligible for a potential seven-year marketing exclusivity for Desidustat.
Looking Ahead
The company sees this designation as an important step in addressing the medical need for treatments for beta-thalassemia.
Source: BSE
