Sickle cell disease: Zydus Lifesciences said it has received Orphan Drug Designation from the US Food and Drug Administration for desidustat, an oral drug candidate being developed for the treatment of sickle cell disease.
Orphan Drug Designation is intended to encourage the development of treatments for rare diseases affecting fewer than 200,000 people in the United States, and can unlock incentives that support clinical development.
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Zydus said the designation highlights the need for additional therapies for sickle cell disease, where treatment options remain limited. The company believes desidustat could help address this unmet need.
The company said it has completed a Phase II, double-blind, randomised, placebo-controlled, multi-centre proof-of-concept study evaluating the efficacy and safety of desidustat tablets in sickle cell disease, and that the data will be published in a medical journal.
Under the FDA’s orphan drug programme, the designation may make the drug eligible for benefits such as tax credits for qualified clinical testing, certain fee waivers, and the possibility of seven years of market exclusivity in the U.S. if the product is approved.
