Zydus today announced that the US Food and Drug Administration has granted Orphan Drug Designation to Desidustat for the treatment of sickle cell disease. Desidustat is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor. The USFDA Office of Orphan Drug Products grants orphan status to medicines intended for rare diseases affecting fewer than 200,000 people in the United States.
Commenting on the development, Dr Sharvil Patel, Managing Director, 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.”
Treatment options for sickle cell disease remain limited. Hydroxyurea has reduced the frequency of painful crises in patients with sickle cell disease, but it is not effective in all patients and is associated with side effects including neutropenia and thrombocytopenia. Blood transfusions involve high costs, limited access, and risks such as alloimmunization, haemolysis, and transfusion-related iron overload.
A Phase II, double blind, randomised, placebo controlled, parallel, multi-centre proof-of-concept study evaluating the efficacy and safety of Desidustat oral tablets for the treatment of sickle cell disease has been completed. Data from the study will be published in a medical journal.
The orphan drug designation for Desidustat makes the programme eligible for development incentives from the USFDA. These include tax credits for qualified clinical testing, exemption from prescription drug user fees, and the possibility of seven years of market exclusivity following regulatory approval.