The approval boosts standard of care for rare disease patients in the country, marking a significant progress this upcoming Rare Disease Day 2025
Bengaluru, January 16, 2025: AstraZeneca Pharma India Limited, a science-led biopharmaceutical company, announces a Central Drugs Standard Control Organisation (CDSCO) permission to import, sale or distribute ‘Eculizumab’ concentrate solution for infusion 300 mg (10mg/ml).
Through this approval, Eculizumab becomes a first anti-complement medical therapy indicated for treating two rare conditions- Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS), both in children and adults.
PNH is a life-threatening disease characterized by thrombosis, end organ damage and impaired quality of life due to chronic haemolysis. Historical global data reveals poor survival rates, with 72% of patients dying within 25 years of diagnosis and without timely diagnosis and treatment options, patients face increased risks of complications such as thrombosis, renal impairment and premature mortality. aHUS on the other hand, presents a grave unmet medical need in India, marked by devastating clinical manifestations, including thrombotic microangiopathy (TMA), acute renal failure, neurological complications, and multi-organ damage. These presentations often result in prolonged hospitalizations, increased healthcare costs, and substantial morbidity.
“With an ambition to be pioneers in science and lead in specialist disease areas, we are proud to bring this innovative therapy for rare disease patients in the country. This is a clear reflection of our purpose to expand access to novel therapies and transform patient outcomes across therapy areas where we believe we can make the most meaningful difference. This approval addresses long standing unmet patient need and offers hope to rare disease community in the country. Following this approval, we remain committed to collaborate with the ecosystem and advance access, putting patients first”, said Dr Sanjeev Panchal, Country President & Managing Director AstraZeneca Pharma India Limited.
AstraZeneca completed the acquisition of Alexion in the year 2021 and continues to expand its portfolio by bringing innovative therapies for patients living with rare diseases in India. Alexion’s pioneering legacy in rare diseases is rooted in being the first to translate the complex biology of the complement system into transformative medicines. By driving innovative research and development (R&D) across new disease targets and modalities, AstraZeneca has diversified its pipeline into additional rare diseases over the last several years. The organisation today is focused on uncovering science and building bridges across our scientific platforms with a focus on bringing more innovative medicines to people worldwide.
About AstraZeneca Pharma India Ltd
AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in four therapy areas - Oncology, Cardiovascular, Renal & Metabolism and Respiratory and Rare Disease. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Established in 1979, AstraZeneca Pharma India Limited is headquartered at Bengaluru, Karnataka in India and has a workforce of over 800 employees across the country committed to deliver great medicines to patients through innovative science and global excellence in development and commercialization. For more information, please visit our website: https://www.astrazeneca.in/ or follow us on LinkedIn: AstraZeneca India.
For further information, please contact AstraZeneca India media relations team:
Faizee Zakir: 7017476868 | faizee.zakir@finnpartners.com