India close to developing drug for sickle cell disease as Zydus-ICMR candidate enters phase III trials

India's efforts to develop an effective treatment for the neglected disease sickle cell anaemia have received a significant boost, with Zydus Lifesciences' drug candidate, Desidustat, advancing to Phase III clinical trials in collaboration with the Indian Council of Medical Research (ICMR).

Sickle cell disease is particularly prevalent among tribal populations, and despite affecting millions of Indians, treatment options have remained limited for decades.

According to the National Health Mission, nearly 20 million people in India live with sickle cell disease, while around 50,000 children are born with the condition every year. Because the disease affects relatively small and economically disadvantaged patient populations, multinational pharmaceutical and biotechnology companies have traditionally shown limited interest in investing in new therapies, making it one of the world's neglected diseases.

The Mayo Clinic describes sickle cell anaemia as a serious inherited blood disorder characterised by extreme fatigue, severe pain episodes, and recurrent infections. The disease is caused by a genetic mutation that transforms normal, round red blood cells into stiff, crescent-shaped "sickle" cells. Unlike healthy red blood cells, which survive for about 120 days, sickle cells break down within 10 to 20 days, leading to chronic anaemia and multiple complications.

Currently available therapies, including hydroxyurea and blood transfusions, have helped improve outcomes but continue to face challenges related to accessibility, variable effectiveness, and long-term risks. Against this backdrop, Desidustat is being evaluated as a potential first-in-class therapy for treating anaemia associated with sickle cell disease.

Under the collaboration, ICMR and Zydus will undertake a 203-day, Phase III clinical study involving 164 patients diagnosed with sickle cell disease to evaluate the efficacy and safety of oral Desidustat tablets in treating anaemia.

The molecule has already established a clinical track record in another indication. Discovered and developed at Zydus' research and development laboratories, Desidustat received approval from the Drug Controller General of India (DCGI) in March 2022 for treating anaemia in patients with chronic kidney disease (CKD), including both dialysis and non-dialysis patients.

In March 2026, China's National Medical Products Administration (NMPA) approved Desidustat for treating renal anaemia in CKD patients. Additionally, the US Food and Drug Administration (FDA) has granted the drug Orphan Drug Designation (ODD) for the treatment of two rare blood disorders - sickle cell disease and beta-thalassaemia.

"We have 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 huge potential for this Indian innovation in addressing this 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.

"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 improving the quality of life for patients," said Sharvil Patel, Managing Director, Zydus Lifesciences.

Sickle cell disease is classified among the Neglected Tropical Diseases (NTDs) - a group of chronic, debilitating conditions caused by viruses, bacteria, parasites, and toxins. Collectively, NTDs affect more than one billion people worldwide, predominantly in impoverished tropical and subtropical regions with limited access to clean water, sanitation, and healthcare.