Zydus Lifesciences announced on Thursday, July 23, that it has received approval from the US Food and Drug Administration (USFDA) to begin a Phase III clinical trial of its investigational drug Desidustat for treating anaemia in patients with sickle cell disease (SCD). The trial will be conducted in collaboration with the Indian Council of Medical Research (ICMR) and represents a significant advancement in addressing this severe health challenge. According to the company's exchange filing, the approval follows the successful completion of a Phase II proof-of-concept trial that met its primary endpoint and demonstrated positive efficacy and safety outcomes.
The 203-day, double-blind, randomised, placebo-controlled, parallel, multicentre study will enrol 164 patients and evaluate the efficacy and safety of Desidustat oral tablets for treating anaemia associated with sickle cell disease. As reported by CNBC TV18, the study showed Desidustat was well tolerated at doses of up to 150 mg, with only mild adverse events reported and no serious adverse events during the trial. The therapy demonstrated a promising trend towards improving haemoglobin levels and higher responder rates compared with placebo, while maintaining a favourable safety profile across all dose groups. Treatment emergent adverse events were low in incidence and comparable across cohorts, comprising nasopharyngitis, polyarthritis and headache, all mild in severity.
According to Zydus Life, the USFDA has granted Orphan Drug Designation to Desidustat for the treatment of both sickle cell disease and beta-thalassaemia. The therapy represents a potential first-in-class treatment opportunity for sickle cell disease, which will now be evaluated further in the Phase III study. Shares of the company were trading at ₹1,125.50 as of 10:45 am on Thursday, with the stock having gained about 28% in the past six months. The company's research centre has previously developed Desidustat, a hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor, which is already approved in India for treating anaemia in patients with chronic kidney disease (CKD) and received approval in China earlier this year for renal anaemia in CKD patients.
Sickle cell disease remains a major public health challenge in India, particularly among tribal populations, with around 20 million people living with the condition in the country, according to estimates from the National Health Mission. As reported by CNBC TV18, approximately 50,000 children are born with sickle cell anaemia each year. While treatments like hydroxyurea and blood transfusions exist, their limited accessibility, inconsistent effectiveness, and associated risks remain significant challenges. Dr Rajiv Bahl, Secretary, Department of Health Research and Director General of ICMR, stated that the successful completion of the Phase II study marked "a significant leap forward for patients who have limited options beyond hydroxyurea." He emphasized the huge potential of this Indian innovation in addressing severe health challenges and highlighted the collaboration's commitment to clinical research through strong public-private partnerships.
