Zydus Life bags USFDA orphan drug tag for Desidustat indicated for sickle cell disease treatment

Zydus Lifesciences Limited on Friday, February 6, said it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (USFDA) for Desidustat for the treatment of sickle cell disease (SCD).

In a press release filed with the exchanges, the company said Desidustat is a novel oral hypoxia inducible factor (HIF)-prolyl hydroxylase inhibitor (PHI) that has the potential to increase haemoglobin levels and red blood cell counts.

The USFDA’s Office of Orphan Drug Products grants orphan status to therapies aimed at treating rare diseases that affect fewer than 200,000 people in the United States.

Zydus said the orphan drug designation makes Desidustat eligible for certain development incentives, including tax credits for qualified clinical trials, exemption from prescription drug user fees, and a potential seven-year marketing exclusivity, subject to USFDA approval.

“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," the company's managing director Dr Sharvil Patel said.

The company noted that current treatment options for SCD are limited. While hydroxyurea can reduce the frequency of painful crises, it is not universally effective and is associated with side effects such as neutropenia and thrombocytopenia. Blood transfusions, another treatment option, are costly, not uniformly accessible, and carry risks including iron overload and alloimmunisation.

Zydus said a Phase II, double-blind, randomised, placebo-controlled, multi-centre proof-of-concept study evaluating the efficacy and safety of Desidustat in sickle cell disease has been completed, with study data expected to be published in a medical journal.

Desidustat is Zydus’ new chemical entity and has previously been studied across multiple indications related to anaemia.

In January, Zydus Lifesciences Ltd. entered into talks to acquire US-based biopharmaceutical company Ardelyx, people familiar with the matter told CNBC-TV18.

According to the sources, Zydus was evaluating options to either acquire a controlling stake in Ardelyx or purchase select key molecules from the company. To partly fund the potential transaction, Zydus Lifesciences was also exploring a fundraise through a qualified institutional placement (QIP), the people cited above said.

Shares of the company were trading 2% down at ₹887.50 as of 1.07 pm on Friday. The stock has declined 11.97% in the past year.