DCGI Gives Go-Ahead for Phase III Trial of Zydus' Desidustat in Sickle Cell Disease
New Delhi: Zydus Lifesciences Limited (Zydus), an innovation-led global lifesciences company, has received permission to conduct a Phase III clinical trial of Desidustat for patients with sickle cell disease.
Conducted in collaboration with the Indian Council of Medical Research (ICMR), the 203-day study will evaluate the efficacy and safety of Desidustat oral tablets in treating anemia. The trial will enrol 164 patients diagnosed with the disease.
Sickle Cell Disease is a significant public health concern in India, especially among tribal populations where prevalence is higher. According to National Health Mission estimates, nearly 20 million people live with the condition, and roughly 50,000 children are born with sickle cell anaemia annually. 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 & Director General, ICMR, said, “We have successfully completed the Phase II study of Desidustat in Sickle Cell Disease in collaborationwith Zydus Lifesciences.This truly 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 severe health challenge. This collaboration reflects our commitment to clinical research through strong public–private partnerships.”
Speaking on this development, Dr Sharvil Patel, Managing Director, Zydus Lifesciences Ltd., 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.”
Zydus and ICMR had earlier completed a Phase II, double blind, randomised, placebo controlled, parallel, multi-centre, proof-of-concept study, co-funded and co-monitored by ICMR-INTENT (Indian National Clinical Trial and Education Network, Clinical Studies and Trial Unit) to evaluate the efficacy and safety of Desidustat oral tablet for treatment of sickle cell disease.
Desidustat is a hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor (PHI) that stimulates endogenous erythropoietin (EPO) production through a mechanism similar to the physiological response to hypoxia. Discovered and developed at Zydus’ research and development laboratories, Desidustat 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), including patients not on dialysis as well as patients on dialysis. In March 2026, Desidustat was also approved by the National Medical Products Administration (NMPA) of China for the treatment of renal anaemia in CKD patients.
The study found that Desidustat was well tolerated up to 150 mg dose, with only minimal adverse events reported. The trial demonstrated a promising trend toward improvement in Hb levels and higher responder rates compared to placebo in patients with SCD. Across the three dose (50 mg, 100 mg and 150 mg), the drug exhibited a favourable safety and tolerability profile. The incidence of treatment-emergent adverse events (TEAEs) was low and comparable across cohorts. The reported TEAEs included nasopharyngitis, polyarthritis, and headache, all of which were mild in severity. No SAEs were reported during the study period. Additionally, no significant differences were observed between treatment arms in laboratory parameters, vital signs, physical examinations, or 12-lead ECG findings.
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