AstraZeneca India has launched Eculizumab concentrate solution for infusion (300 mg,10 mg/ml) in India, following 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 authorised 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 percent 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 ensure these breakthroughs reach those who need them the most, without delay. The launch of Eculizumab as India’s first approved anti-complement therapy demonstrates our dedication to deliver 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 expand our access and make a meaningful difference for people living with rare diseases.”

The launch of Eculizumab in India aligns with AstraZeneca’s ambition to transform outcomes in rare and complex diseases through pioneering science and early intervention.

“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, Department of Paediatric Nephrology, Apollo Hospital, Indraprastha, Delhi.

Through ongoing research and portfolio expansion, AstraZeneca is reinforcing its commitment to address rare and complex conditions in India. By focusing on scientific discovery and innovative therapies, the company aims to bridge gaps in diagnosis and treatment, delivering meaningful benefits to patients and their families.