Zydus Lifesciences has received approval to begin a Phase III clinical trial of Desidustat for the treatment of anaemia in patients with sickle cell disease (SCD), in collaboration with the Indian Council of Medical Research (ICMR).
The 203-day study will enrol 164 patients with sickle cell disease to assess the efficacy and safety of Desidustat oral tablets in treating anaemia, a common and debilitating complication of the condition.
Zydus highlighted that sickle cell disease remains a major public health challenge in India, particularly among tribal communities where its prevalence is significantly higher. According to National Health Mission estimates, nearly 20 million people in the country are living with the disease, while around 50,000 children are born with sickle cell anaemia each year.
Although hydroxyurea therapy and blood transfusions are currently available, access to these treatments remains limited. Additionally, their inconsistent effectiveness and associated risks remain significant challenges, highlighting the need for new treatment options.
Dr. Rajiv Bahl, secretary, Department of Health Research & Director General, ICMR, said that the successful completion of the Phase II study marks “a significant leap forward for patients who have limited options beyond hydroxyurea.”
“As we move towards Phase III trials, we see a huge potential of this Indian innovation in addressing a severe health challenge. This collaboration reflects our commitment to clinical research through strong public–private partnerships,” he added.
Dr. Sharvil Patel, managing director of Zydus Lifesciences said sickle cell disease severely impacts the lives of millions of people and represents a high unmet medical need.
“We are happy to collaborate with ICMR to develop new and effective therapeutic options for patients living with Sickle Cell Disease. Desidustat, discovered and developed at the Zydus Research Centre, reflects our commitment to advancing novel innovations and improve quality of life for patients,” he said.
About Desidustat
Desidustat is a hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) that promotes the body’s natural production of erythropoietin (EPO), mimicking the physiological response to low oxygen levels (hypoxia).
Discovered and developed at Zydus’ research and development laboratories, the drug received approval from the Drug Controller General of India (DCGI) in March 2022 for the treatment of anaemia in patients with chronic kidney disease (CKD), in both dialysis-dependent and non-dialysis patients. In March 2026, China’s National Medical Products Administration (NMPA) also approved Desidustat for the treatment of renal anaemia in CKD patients.
The Phase III study follows encouraging results from an earlier Phase II proof-of-concept trial jointly conducted by Zydus and ICMR under the ICMR-INTENT (Indian National Clinical Trial and Education Network) programme.
The Phase II trial showed that Desidustat was well tolerated at doses of up to 150 mg, with only minimal adverse events reported. The study also demonstrated a positive trend towards improved haemoglobin levels and higher response rates compared with placebo in patients with SCD, while maintaining a favourable safety and tolerability profile across all three dose levels (50 mg, 100 mg and 150 mg).
