Zydus Lifesciences Limited Secures Approval for Phase III Desidustat Trial with ICMR
July 23, 2026: Zydus Lifesciences Limited has received approval to initiate a Phase III clinical trial to evaluate Desidustat for the treatment of anemia associated with Sickle Cell Disease (SCD). The study will be conducted in collaboration with the Indian Council of Medical Research (ICMR), marking an important milestone in the company’s efforts to address a significant unmet medical need in India.
The approval follows encouraging results from an earlier Phase II clinical study and strengthens Zydus’ clinical pipeline for Desidustat, an innovative oral therapy developed by the company’s research team.
Phase III Trial to Evaluate Safety and Effectiveness
The Phase III study has been designed as a double-blind, randomized, placebo-controlled, parallel-group, multicenter clinical trial, considered the gold standard for evaluating the safety and efficacy of new medicines.
Key Highlights of the Trial
- Drug: Desidustat
- Indication: Treatment of anemia in patients with Sickle Cell Disease (SCD)
- Study Design: Double-blind, randomized, placebo-controlled, parallel-group, multicenter trial
- Study Duration: 203 days
- Participants: 164 patients with Sickle Cell Disease across India
- Collaborating Organization: Indian Council of Medical Research (ICMR)
The trial aims to assess whether oral Desidustat can safely improve hemoglobin levels and reduce anemia-related complications in patients living with Sickle Cell Disease.
Encouraging Results from Phase II Study
The Phase III trial follows positive findings from the earlier Phase II proof-of-concept study, which was co-funded and co-monitored by ICMR-INTENT.
According to the company:
- Desidustat was well tolerated at doses of up to 150 mg.
- Patients receiving the drug demonstrated a higher response rate compared with placebo.
- The treatment showed encouraging improvements in hemoglobin (Hb) levels.
- Most treatment-emergent adverse events were mild, indicating a favorable safety profile.
These results provided the scientific basis for advancing the therapy into a larger Phase III study.
Global Recognition for Desidustat
Desidustat has already received important regulatory recognition internationally.
The drug has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of:
- Sickle Cell Disease (SCD)
- Beta-thalassemia
Orphan Drug Designation is awarded to therapies intended to treat rare diseases and can provide regulatory and development incentives.
Existing Regulatory Approvals
Desidustat is not a new molecule. It has already been approved for the treatment of renal anemia in patients with chronic kidney disease (CKD).
The therapy received:
- Approval in India from the Central Drugs Standard Control Organization (CDSCO) in March 2022
- Approval in China from the National Medical Products Administration (NMPA) in March 2026
The drug was discovered and developed at Zydus Research Centre and belongs to a class of medicines known as Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitors (HIF-PHIs).
Addressing a Major Public Health Challenge
Sickle Cell Disease remains one of India’s significant inherited blood disorders, affecting an estimated 20 million people, with nearly 50,000 babies born with the condition every year.
Patients frequently suffer from chronic anemia, painful crises, organ complications, and reduced quality of life. Current treatment options are limited and often include hydroxyurea therapy, blood transfusions, and supportive care.
If successful, Desidustat could offer a first-in-class oral treatment option for managing anemia associated with Sickle Cell Disease, potentially reducing dependence on repeated blood transfusions and improving patient outcomes.