Zydus Lifesciences Receives Approval to Launch Phase III Trial of Desidustat for Sickle Cell Disease

Zydus Lifesciences Receives Approval to Launch Phase III Trial of Desidustat for Sickle Cell Disease​

Zydus Lifesciences Limited has received permission to initiate a Phase III clinical trial of its drug, Desidustat, aimed at treating patients with sickle cell disease (SCD). The 203-day study will be conducted in collaboration with the Indian Council of Medical Research (ICMR) and is set to enroll 164 patients diagnosed with the condition.

The Phase III trial is designed as a double blind, randomized, placebo controlled, parallel, multicentre study to assess the efficacy and safety of Desidustat oral tablets for treating anemia in SCD patients. This drug represents a potential first-in-class therapeutic option for managing sickle cell disease, following successful completion of earlier studies.

Zydus Lifesciences Limited, an innovation-led global lifesciences company, is advancing its commitment to developing novel treatments for unmet healthcare needs. The approval comes after Zydus and ICMR successfully completed a Phase II Proof-of-concept (PoC) trial evaluating the efficacy and safety of Desidustat oral tablet for sickle cell disease.

Clinical Trial Background and Significance​

Sickle Cell Disease poses a major public health challenge in India, particularly among tribal populations where prevalence is high. National Health Mission estimates indicate that approximately 20 million people live with SCD, and around 50,000 children are born with sickle cell anaemia each year. Current treatments, including blood transfusions and hydroxyurea, face limitations regarding accessibility, consistency of effectiveness, and associated risks.

Dr. Rajiv Bahl, Secretary of the Department of Health Research and Director General at ICMR, noted that the successful completion of the Phase II study marks a significant development for patients who have limited options beyond standard care. He emphasized that this collaboration reflects a commitment to clinical research through public-private partnerships.

Dr. Sharvil Patel, Managing Director at Zydus Lifesciences Ltd., stated that addressing sickle cell disease is crucial due to the severe impact it has on millions of people and represents a high unmet medical need. Desidustat, which was discovered and developed at the Zydus Research Centre, reflects the company's commitment to advancing novel innovations and improving the quality of life for patients.

Pre-clinical Findings and Drug Profile​

Prior to the Phase III study, Zydus and ICMR conducted a Phase II study—a double blind, randomized, placebo controlled, parallel, multi-centre, proof-of-concept trial—to evaluate Desidustat. This initial study demonstrated that the drug was well tolerated up to a 150 mg dose, exhibiting a favorable safety and tolerability profile across three dosage cohorts (50 mg, 100 mg, and 150 mg).

The Phase II findings showed a promising trend toward improvement in hemoglobin levels and higher responder rates compared to the placebo group among patients with SCD. Treatment emergent adverse events (TEAEs) reported included nasopharyngitis, polyarthritis, and headache, all of which were mild in severity. No serious adverse events (SAEs) were reported during this period.

Desidustat is classified as a hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor (PHI). This drug stimulates endogenous erythropoietin (EPO) production through a mechanism similar to the body's physiological response to hypoxia. Desidustat has received Orphan Drug Designation (ODD) from the US FDA for the treatment of two rare blood disorders: SCD and beta-thalassemia.

In addition, Desidustat obtained approval from the Drug Controller General of India (DCGI) in March 2022 for treating anaemia in patients with chronic kidney disease (CKD), covering both those on and not on dialysis. It was also approved by the Medical Products Administration (NMPA) of China in March 2026 for renal anemia in CKD patients.

ZYDUSLIFE Stock Price Movement​

As of 10:07 AM, shares of Zydus Lifesciences Limited are slipping by 0.63% in live trading, currently trading at ₹1119.2. The stock has seen a volume of 103,554 shares as the session progresses.