AstraZeneca’s recent announcement of the launch of eculizumab in India marks a significant milestone in the treatment of rare diseases.

For patients suffering from ultra-rare, life-threatening disorders such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), this launch brings hope to the Indian medical community for access to long-awaited therapies.

Regulatory Approval & Launch in India

CDSCO Approval

• In January 2025, the Central Drugs Standard Control Organisation (CDSCO) in India approved Eculizumab concentrate solution for infusion (300 mg, 10 mg/mL) for import, sale, and distribution.
• The approval covers use in both adults and children, for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS).

Official Launch

• AstraZeneca India officially launched Eculizumab in September 2025. The launch brings it into the Indian market as the first anti-complement therapy authorised in India for PNH and aHUS.
• The introduction of Eculizumab in India represents a major step forward in the country’s rare disease ecosystem.

Indications: What Conditions Does Eculizumab Treat?

• Paroxysmal Nocturnal Hemoglobinuria (PNH): A rare disease in which red blood cells are destroyed (hemolysis), leading to anemia, fatigue, increased risk of thrombosis (blood clots), and organ damage if left untreated. Worldwide, without treatment, a large proportion of PNH patients have a poor chance of long-term survival.
• Atypical Hemolytic Uremic Syndrome (aHUS): An ultra-rare disease involving uncontrolled activation of the complement system, resulting in thrombotic microangiopathy (TMA), acute kidney damage, neurological effects, and possibly multiple organ damage. In India, the prevalence of aHUS is estimated to be low (0.23-1.9 cases per million per year), but it has serious consequences.

Both these indications have now been formally approved for eculizumab in India. This has expanded the treatment options for patients suffering from these serious disorders.

Why the Launch of Eculizumab in India Matters

Filling a Critical Unmet Need

• Prior to this launch, patients in India had limited or no access to authorized anti-complement therapy for PNH/aHUS. Delays in diagnosis and the lack of effective treatment resulted in high rates of morbidity, hospitalization, and complications such as kidney failure, thrombosis, and organ damage.
• With eculizumab in India, the path has now been paved for advanced, targeted therapy instead of just supportive management (dialysis, blood transfusion, etc.). This can significantly improve quality of life and outcomes.

Impact on the Rare Disease Community

• Policies related to rare diseases are evolving in India (e.g., increased funding, increased awareness). The CDSCO approval and the launch of eculizumab reinforce that rare diseases are being taken more seriously from a policy and regulatory perspective.
• For patients and their families, this launch offers hope for survival and reduced long-term complications.

AstraZeneca’s Role & Global Perspective

• AstraZeneca acquired Alexion (developer of Eculizumab) in 2021. This acquisition brought into their portfolio several rare-disease therapies.
• Globally, Soliris (Eculizumab) is a leading rare disease asset. In 2024 its net worldwide sales were approx US$2.58 billion.
• India now becomes part of the market where this advanced therapy is available via formal regulatory approval, rather than via import on special requests.

What the Future Holds

The launch of Eculizumab in India could have several ripple effects:

• Encourage improved protocols for early detection of complement-mediated diseases.
• Encourage the development of local guidelines for use, monitoring, and follow-up.
• Encourage clinical trials or data collection for rare complement disorders in India.
• Further advance rare disease policy (funding, insurance coverage, patient assistance programs).

Conclusion

The launch of eculizumab in India by AstraZeneca is a landmark achievement in rare disease care. It means that for the first time, a scientifically proven, targeted anti-complement therapy for PNH and aHUS in both adults and children is officially available under Indian regulations.

While challenges such as cost, diagnostics and infrastructure still remain, this is an important first step. For patients, families and healthcare providers dealing with rare diseases, eculizumab in India brings hope and a new standard of care.

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