USFDA Grants Orphan Drug Status to Zydus' Desidustat for Sickle Cell Disease
New Delhi: Zydus Lifesciences Limited on Wednesday announced that the United States Food and Drug Administration (USFDA) has granted Orphan Drug Designation (ODD) to its novel New Chemical Entity (NCE) Desidustat for the treatment of Sickle Cell Disease (SCD), a rare and debilitating genetic blood disorder.
The designation has been granted by the USFDA’s Office of Orphan Drug Products, which supports the development of therapies for rare diseases affecting fewer than 200,000 patients in the United States.
Desidustat is an oral hypoxia inducible factor prolyl hydroxylase inhibitor (HIF-PHI) with the potential to increase haemoglobin levels and red blood cell counts. The drug is being evaluated as a novel therapeutic option for patients with sickle cell disease, where treatment choices remain limited and are often associated with safety, accessibility, and long-term efficacy concerns.
Commenting on the development, Dr. Sharvil Patel, Managing Director of Zydus Lifesciences Limited, said, “This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop a therapy for sickle cell disease. We believe that Desidustat can address this unmet need.”
Currently available therapies for sickle cell disease have significant limitations. While hydroxyurea has been shown to reduce the frequency of painful crises, it is not universally effective and is associated with adverse effects such as neutropenia and thrombocytopenia. Blood transfusions, another key management strategy, are costly, not uniformly accessible, and carry risks including alloimmunization, hemolysis, and iron overload.
Zydus has completed a Phase II, double-blind, randomized, placebo-controlled, parallel, multi-centre proof-of-concept study to evaluate the efficacy and safety of Desidustat oral tablets in patients with sickle cell disease. The company stated that data from the study will be published in a peer-reviewed medical journal.
The orphan drug designation makes Desidustat eligible for several regulatory and financial incentives, including tax credits for qualified clinical trials, exemption from prescription drug user fees, and a potential seven-year period of market exclusivity in the US, subject to regulatory approval
Zydus Lifesciences Limited is a global life sciences company engaged in the discovery, development, manufacturing, and marketing of a wide range of healthcare products. With a workforce of over 29,000 employees worldwide, including more than 1,500 scientists dedicated to research and development, the company has introduced several first-in-class and innovative products over the past decade across vaccines, therapeutics, biologics, and new chemical entities, targeting areas of unmet medical need.
