Zydus’ Desidustat gets Orphan Drug Designation from U.S. FDA for Sickle Cell disease

Zydus on Friday said U.S. Food and Drug Administration has granted Orphan Drug Designation (ODD) to Desidustat for the treatment of Sickle Cell Disease (SCD).

The ODD 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 U.S. FDA approval.

Zydus’ NCE Desidustat is a hypoxia inducible factor (HIF)-prolyl hydroxylase inhibitor (PHI) and has the potential to increase haemoglobin and red blood cell counts, the company said. The U.S. FDA’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 U.S.

The ODD underlines the urgent medical need to develop a therapy for sickle cell disease. “We believe Desidustat can address this unmet need,” Zydus Lifesciences Managing Director Sharvil Patel said. 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 said.