Zydus has received Orphan Drug Designation (ODD) from the USFDA for Desidustat, a novel oral HIF-PHI, to treat Sickle Cell Disease (SCD). The ODD provides eligibility for a potential seven-year marketing exclusivity upon USFDA approval. Desidustat may address the urgent medical need for SCD treatment and offer new hope for patients. Clinical trials have already shown promising results.
Orphan Drug Designation Received
Zydus announced that the USFDA has granted Orphan Drug Designation (ODD) to Desidustat for the treatment of Sickle Cell Disease (SCD). Desidustat, a hypoxia inducible factor (HIF)-prolyl hydroxylase inhibitor (PHI), has the potential to increase haemoglobin and red blood cell counts. The designation was announced on February 6, 2026.
Benefits of ODD Status
The ODD by the USFDA for Desidustat provides eligibility for a potential seven-year marketing exclusivity. It also includes tax credits for qualified clinical testing and prescription drug user fee exemptions.
Desidustat’s Potential Impact
Desidustat, if approved, could offer a new treatment option for SCD. Dr. Sharvil Patel, Managing Director, Zydus Lifesciences Limited, stated that the ODD “underlines the urgent medical need to develop a therapy for sickle cell disease” and believes that Desidustat can address this unmet need.
Clinical Development
A Phase II, double-blind, randomized, placebo-controlled, parallel, multi-centre, proof-of-concept study to evaluate the efficacy and safety of Desidustat oral tablet for treatment of SCD has been completed, and data will be published in medical journal. The company has completed a study and plans to release data soon.
About Desidustat
Desidustat is a hypoxia inducible factor (HIF)-prolyl hydroxylase inhibitor (PHI) with the potential to increase haemoglobin and red blood cell counts.
Source: BSE
