Zydus Lifesciences Secures USFDA Orphan Drug Designation for Desidustat
Filing Summary
Zydus Lifesciences Ltd has received the USFDA Orphan Drug Designation for its novel oral drug, Desidustat, aimed at treating Sickle Cell Disease. This designation provides eligibility for potential seven-year marketing exclusivity, subject to USFDA approval. Desidustat is a hypoxia inducible factor-prolyl hydroxylase inhibitor, which may increase haemoglobin and red blood cell counts. The USFDA grants orphan status to drugs that treat rare diseases affecting fewer than 200,000 people in the United States. A Phase II study evaluating Desidustat’s efficacy and safety has been completed.
Zydus Lifesciences Ltd has announced that the United States Food and Drug Administration (USFDA) has granted Orphan Drug Designation (ODD) to its novel oral drug, Desidustat, for the treatment of Sickle Cell Disease (SCD). This designation is intended to support the development of drugs for rare diseases affecting fewer than 200,000 individuals in the United States. Desidustat is classified as a hypoxia inducible factor-prolyl hydroxylase inhibitor (HIF-PHI), which may potentially increase haemoglobin and red blood cell counts.
The Orphan Drug Designation by the USFDA provides eligibility for certain development incentives. These include tax credits for qualified clinical testing, exemptions from prescription drug user fees, and a potential seven-year marketing exclusivity upon USFDA approval. The designation does not imply any immediate financial impact but offers strategic advantages in the drug development process.
Desidustat is designed to address the therapeutic needs in the management of Sickle Cell Disease, where current options are limited. The drug has undergone a Phase II, double-blind, randomized, placebo-controlled, parallel, multi-centre, proof-of-concept study to evaluate its efficacy and safety. The results from this study will be published in a medical journal. The drug’s mechanism involves inhibiting prolyl hydroxylase, which may help in increasing haemoglobin levels and reducing the frequency of painful crises associated with SCD.
The USFDA’s Orphan Drug Designation is part of a broader regulatory framework aimed at encouraging the development of treatments for rare diseases. This framework provides various incentives to pharmaceutical companies to invest in research and development for conditions that have limited treatment options. The designation for Desidustat aligns with Zydus Lifesciences’ strategic focus on developing innovative therapies for unmet medical needs.
The timeline for the development and potential commercialization of Desidustat includes the completion of a Phase II study, with further clinical trials likely required before seeking full regulatory approval. The company has not disclosed specific dates for subsequent phases or anticipated approval timelines. The designation marks a significant milestone in the drug’s development pathway, providing a structured framework for its advancement.
Zydus Lifesciences Ltd is a global pharmaceutical company focused on discovering, developing, and marketing a broad range of healthcare therapies. The company employs over 29,000 individuals worldwide, including 1,500 scientists dedicated to research and development. Zydus Lifesciences is committed to unlocking new possibilities in life sciences through quality healthcare solutions and has introduced several innovative products in the market over the past decade.