AstraZeneca India launches Eculizumab, first anti-complement therapy for aHUS & PNH

New Delhi: AstraZeneca India has officially launched Eculizumab concentrate solution for infusion (300 mg,10 mg/ml) in India. This follows the Central Drugs Standard Control Organisation (CDSCO) approval received in January 2025 for the import, sale, and distribution of the product.

With this development, Eculizumab is now the first anti-complement therapy authorized in India for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS) in both adults and children.

Both aHUS and PNH are ultra-rare, life-threatening disorders caused by chronic, uncontrolled activation of the complement system, leading to severe complications in blood and kidney health.

aHUS is an ultra-rare, progressive disorder triggered by, uncontrolled activation of the complement system, leading to thrombotic microangiopathy (TMA)- the formation of blood clots in small vessels. According to a study by BMC Nephrology, aHUS occurs at rates as low as 0.23–1.9 cases per million annually in India, with root causes ranging from pregnancy and infections to certain cancers. Its clinical course may result in kidney failure, haemolytic anaemia, high blood pressure, and, in severe cases, heart disease or stroke.

PNH is equally serious, with a global median survival of just 10–20 years ^]without treatment. Thrombosis is the leading cause of death, with up to 72% of untreated patients dying within 25 years of diagnosis.

Until now, limited awareness, diagnosis, and access to targeted therapies have contributed to prolonged hospitalisations and increased complications for those affected. The availability of Eculizumab marks a breakthrough in the standard of care.

Praveen Akkinepally, Country President and Managing Director, AstraZeneca India, said, “Science has the power to redefine what’s possible for patients, and at AstraZeneca, we are deeply committed to ensuring these breakthroughs reach those who need them most, without delay. The launch of Eculizumab as India’s first approved anti-complement therapy demonstrates our dedication to delivering innovative medicines at the earliest opportunity, especially where there is significant unmet medical need. By bringing forward transformative therapies, we strive to give patients in India timely access to advances that can change lives and improve long-term outcomes. This milestone reflects our continued commitment to expanding our access and making a meaningful difference for people living with rare diseases.”

“The availability of Eculizumab in India is a transformative step for patients with PNH and aHUS. These diseases are often not promptly diagnosed and have lacked targeted treatment options. Having access to a proven anti-complement therapy will significantly improve patient prognosis, reduce complications, and help prevent irreversible organ damage” said, Dr Arvind Bagga, Senior Consultant, Dept. of Paediatric Nephrology, Apollo Hospital, Indraprastha, Delhi.