Zydus, ICMR Receive Approval to Begin Phase III Trial of Desidustat for Sickle Cell Disease

Ahmedabad, July 23, 2026: Zydus Lifesciences Limited has received regulatory approval to conduct a Phase III clinical trial of Desidustat for the treatment of anaemia in patients with sickle cell disease (SCD). The study will be carried out in collaboration with the Indian Council of Medical Research (ICMR) and follows the successful completion of a Phase II proof-of-concept (PoC) trial that demonstrated encouraging efficacy and safety outcomes.

The 203-day Phase III study will be a double-blind, randomised, placebo-controlled, parallel, multicentre trial designed to evaluate the efficacy and safety of Desidustat oral tablets in treating anaemia among patients with sickle cell disease. The trial is expected to enrol 164 patients diagnosed with the condition.

The development marks another milestone for Desidustat, which has also received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of two rare blood disorders—sickle cell disease and beta-thalassemia. If successful, Desidustat could emerge as a potential first-in-class therapeutic option for patients living with sickle cell disease.

Addressing a Major Public Health Challenge

Sickle cell disease remains a significant public health concern in India, particularly among tribal communities where the prevalence is considerably higher. According to National Health Mission estimates, nearly 20 million people in India live with the condition, while approximately 50,000 children are born with sickle cell anaemia every year.

Although treatment options such as hydroxyurea therapy and blood transfusions are available, accessibility issues, inconsistent effectiveness and associated risks continue to pose major challenges, highlighting the need for newer treatment approaches.

"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. This collaboration reflects our commitment to clinical research through strong public–private partnerships," said Dr. Rajiv Bahl, Secretary, Department of Health Research and Director General, ICMR.

"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," said Dr. Sharvil Patel, Managing Director, Zydus Lifesciences Ltd.

Phase II Trial Demonstrated Promising Results

The Phase II proof-of-concept study, jointly conducted by Zydus and ICMR, was a double-blind, randomised, placebo-controlled, parallel, multicentre clinical trial. The study was co-funded and co-monitored by ICMR-INTENT (Indian National Clinical Trial and Education Network, Clinical Studies and Trial Unit) to assess the efficacy and safety of Desidustat oral tablets in patients with sickle cell disease.

The study achieved its primary endpoint and found that Desidustat was well tolerated at doses of up to 150 mg. The trial demonstrated a promising trend towards improved haemoglobin (Hb) levels and higher responder rates compared to placebo. Across the evaluated doses of 50 mg, 100 mg and 150 mg, the therapy maintained a favourable safety and tolerability profile.

The incidence of treatment-emergent adverse events (TEAEs) remained low and was comparable across all treatment groups. Reported adverse events included nasopharyngitis, polyarthritis and headache, all of which were mild in severity. No serious adverse events (SAEs) were reported during the study. Researchers also observed no significant differences between treatment groups in laboratory parameters, vital signs, physical examinations or 12-lead ECG findings.

The completed clinical study has been registered under CTRI Registration: CTRI/2024/06/068363.

About Zydus Lifesciences

Zydus Lifesciences Limited is a global innovation-driven life sciences company engaged in discovering, developing, manufacturing and marketing a broad portfolio of healthcare therapies. The company focuses on advancing novel treatments across multiple therapeutic areas through research and clinical innovation.

 

(Inputs from Press Release)