Clinical Trial Approval Details
Zydus Lifesciences Limited has received permission to conduct a Phase III clinical trial of Desidustat for patients with sickle cell disease. The trial will be 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 in sickle cell disease patients. The trial will enroll 164 patients diagnosed with the disease.
Phase II Trial Results
Zydus and ICMR successfully completed a Phase II, double blind, randomized, 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). 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 doses (50 mg, 100 mg and 150 mg), the drug exhibited a favorable safety and tolerability profile.
Safety 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. [CTRI Registration: CTRI/2024/06/068363]
Trial Design
The Phase III trial will be a double blind, randomized, placebo controlled, parallel, multicentre study to evaluate the efficacy and safety of Desidustat oral tablets for treatment of anemia in sickle cell disease patients.
Regulatory Status
The US FDA has granted Orphan Drug Designation (ODD) to Zydus' Desidustat for treating two rare blood disorders: Sickle Cell Disease (SCD) and beta-thalassemia. Desidustat received approval from the Drug Controller General of India (DCGI) in March 2022 for the treatment of anemia 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 anemia in CKD patients.
Drug Mechanism
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. The drug was discovered and developed at Zydus' research and development laboratories.
Disease Context
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 anemia annually. While treatments like hydroxyurea and blood transfusions exist, their limited accessibility, inconsistent effectiveness, and associated risks remain significant challenges.
Management Commentary
Dr. Rajiv Bahl, Secretary, Department of Health Research & Director General, ICMR, stated: \"We have successfully completed the Phase II study of Desidustat in Sickle Cell Disease in collaboration with 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.\"
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.\"
References
The announcement includes 16 scientific references supporting the development and clinical evaluation of Desidustat across various studies and publications.