Zydus Lifesciences Gets Approval for Phase 3 Trial of Desidustat in Sickle Cell Disease

Study to evaluate oral desidustat in patients with sickle cell disease and anemia

Zydus Lifesciences has received permission to conduct a Phase 3 clinical trial of desidustat in patients with sickle cell disease in collaboration with the Indian Council of Medical Research (ICMR).

The 203-day study will evaluate the efficacy and safety of oral desidustat tablets in 164 patients diagnosed with anemia associated with sickle cell disease.

The clinical trial is being planned as the company looks to evaluate a new treatment approach for patients who continue to face limited options for managing anemia linked to sickle cell disease.

Sickle Cell Disease Remains a Major Health Challenge in India

Sickle cell disease is a genetic blood disorder that can severely affect a person's health and quality of life.

In India, the condition continues to affect a large number of people. According to estimates cited by the National Health Mission, around 50,000 children are born with sickle cell anemia every year, while nearly 20 million people are living with the condition.

The disease affects red blood cells and can lead to anemia, pain episodes and other serious health complications.

For many patients, managing the condition over the long term can be difficult.

Current Treatment Options Still Have Challenges

Existing treatments for sickle cell disease include medicines such as hydroxyurea and supportive approaches such as blood transfusions.

However, these options do not work equally well for every patient and can come with their own challenges and risks.

This has created a need for additional treatment options that can help manage anemia and improve the lives of people living with sickle cell disease.

Zydus is now evaluating whether desidustat could become one such option.

How Desidustat Could Help Patients

Desidustat works differently from traditional injectable treatments used to stimulate red blood cell production.

The drug is designed to activate a natural process that the body normally uses when it senses low oxygen levels.

It works by stabilizing proteins called hypoxia-inducible factors, or HIFs.

These proteins help stimulate the body's natural production of erythropoietin, also known as EPO. EPO is a hormone that plays an important role in the production of red blood cells.

By preserving HIF activity, desidustat aims to mimic the body's natural response to low oxygen levels.

This process may help increase natural EPO levels and improve the body's use of iron, which is needed to produce healthy red blood cells.

Because desidustat is available as an oral tablet, the treatment could also offer an alternative to some traditional injectable therapies if its effectiveness and safety are confirmed in clinical trials.

Phase 2 Study Showed Promising Hemoglobin Results

Before moving into Phase 3 development, desidustat was evaluated in a Phase 2 multi-center study in patients with sickle cell disease.

According to Zydus, the 150 mg dose showed promising improvements in hemoglobin levels.

The company also reported that only minimal adverse events were observed during the study.

These findings have supported the decision to move forward with a larger Phase 3 clinical trial.

The new study will provide more data on the drug's potential benefits and safety in a broader group of patients.

ICMR and Zydus Continue Clinical Development

The Phase 3 trial is being conducted in collaboration with ICMR.

Dr Rajiv Bahl, Secretary and Director General of ICMR, said the successful completion of the Phase 2 study represents an important step for patients who have limited treatment options beyond hydroxyurea.

The collaboration between Zydus and ICMR is expected to support the next stage of clinical development for desidustat in sickle cell disease.

The Phase 3 study will focus on 164 patients and will run for 203 days.

Researchers will assess the efficacy and safety of oral desidustat tablets in patients with anemia associated with sickle cell disease.

Desidustat Is Already Approved for CKD-Related Anemia in India

Desidustat is not a completely new medicine.

In India, the drug is already approved for the treatment of anemia in patients with chronic kidney disease, including patients who are undergoing dialysis.

The drug has also received approval in China from the National Medical Products Administration for the same chronic kidney disease-related anemia indication.

The company's experience with desidustat in other anemia settings has helped support its continued investigation in sickle cell disease.

Zydus Looks to Expand the Use of Its Drug

Desidustat was discovered and developed at the Zydus Research Centre.

Zydus Lifesciences managing director Sharvil Patel said sickle cell disease has a major impact on millions of people and that the development of desidustat reflects the company's focus on developing new treatment options for patients.

The Phase 3 study will now provide further information about whether desidustat can safely improve anemia in people with sickle cell disease and potentially offer patients another oral treatment option.

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