Zydus receives USFDA Orphan Drug Designation for Desidustat to treat Sickle Cell Disease

New Delhi: Zydus, a leading, discovery-based, global pharmaceutical company has announced that the USFDA has granted Orphan Drug Designation (ODD) to Desidustat, a novel oral HIF-PHI, for the treatment of Sickle Cell Disease (SCD).

The USFDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States.

Speaking 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.”

Therapeutic options for management of Sickle Cell Disease are limited. Hydroxyurea, reduced frequency of painful crisis in SCD, but is not universally effective and associated with side-effects like neutropenia and thrombocytopenia. Blood transfusions are expensive, not uniformly accessible, and are accompanied by risks including alloimmunization, hemolysis, and transfusion iron overload. 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.

Orphan drug designation by the USFDA for Desidustat, 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 the USFDA approval.