Zydus Lifesciences Limited has received regulatory approval to begin a Phase III clinical trial of its innovative drug, Desidustat, for the treatment of anaemia in patients with sickle cell disease (SCD). The study will be conducted in collaboration with the Indian Council of Medical Research (ICMR) and marks an important step in the development of a new treatment option for one of India’s most widespread inherited blood disorders.
The 203-day Phase III trial will enrol 164 patients diagnosed with sickle cell disease to evaluate the efficacy and safety of Desidustat oral tablets in treating anaemia associated with the condition. The collaborative study builds on encouraging results from an earlier Phase II clinical trial jointly conducted by Zydus and ICMR.
Sickle cell disease remains a major public health challenge in India, particularly among tribal communities where the disorder is more common. According to estimates under the National Health Mission, nearly 20 million people in India are living with the disease, while around 50,000 children are born with sickle cell anaemia every year. Although therapies such as hydroxyurea and regular blood transfusions are available, many patients continue to face limited access to treatment, inconsistent outcomes, and long-term complications.
Dr Rajiv Bahl, Secretary, Department of Health Research and Director General of ICMR, said the successful completion of the Phase II study has created a strong foundation for the next stage of clinical development. He noted that Desidustat has shown significant promise as a potential treatment for patients who currently have limited therapeutic options beyond hydroxyurea. He added that the partnership between ICMR and Zydus highlights the importance of public-private collaboration in advancing clinical research and developing innovative healthcare solutions for India.
Dr Sharvil Patel, Managing Director of Zydus Lifesciences, said sickle cell disease continues to affect millions of people and represents a major unmet medical need. He said the collaboration with ICMR reflects the company’s commitment to developing new treatment options that can improve patient outcomes and quality of life. He added that Desidustat was discovered and developed at the Zydus Research Centre and represents the company’s continued investment in scientific innovation.
The Phase III study follows a successful Phase II proof-of-concept trial that evaluated three dosage levels of Desidustat—50 mg, 100 mg, and 150 mg. The earlier study found that the drug was well tolerated across all dose levels and demonstrated encouraging improvements in haemoglobin levels compared with placebo.
Researchers reported only a small number of mild treatment-related adverse events, including nasopharyngitis, polyarthritis, and headache. Importantly, no serious adverse events were observed during the study period. Laboratory investigations, vital signs, physical examinations, and ECG assessments also remained comparable across treatment groups, confirming the favourable safety profile of the drug. If the Phase III trial confirms the positive findings seen in earlier studies, Desidustat could become an important new treatment option for patients living with sickle cell disease, offering a convenient oral therapy to address anaemia and improve disease management.
Zydus Lifesciences is a global innovation-driven healthcare company engaged in the discovery, development, manufacturing, and marketing of medicines, vaccines, biologics, and novel therapies. With a workforce of over 30,000 employees, including nearly 1,500 research scientists, the company continues to invest in advanced medical research aimed at addressing unmet healthcare needs and improving patient care worldwide.
