​​Zydus secures USFDA orphan drug tag for sickle cell therapy Desidustat

Zydus Lifesciences has received Orphan Drug Designation from the US Food and Drug Administration (USFDA) for Desidustat, its novel oral therapy being developed for the treatment of Sickle Cell Disease (SCD), the company said on Friday.

The USFDA grants orphan status to medicines intended to treat rare conditions affecting fewer than 200,000 people in the United States, with the aim of encouraging research and development in areas of high unmet medical need.

Commenting on the development, Zydus Lifesciences Managing Director Sharvil Patel said the designation highlights the critical need for new treatment options for patients with sickle cell disease. He added that the company believes Desidustat has the potential to address gaps in current therapies, which remain limited.

Zydus also said it has successfully completed a Phase II, double-blind, randomised, placebo-controlled, multi-centre proof-of-concept study to assess the efficacy and safety of Desidustat in patients with SCD. The clinical data from the study is expected to be published in a peer-reviewed medical journal.

The orphan drug status makes Desidustat eligible for several regulatory incentives, including tax credits for clinical trials, exemption from certain USFDA user fees, and the possibility of seven years of market exclusivity in the US following regulatory approval.

Source: PTI