AstraZeneca expands its portfolio by bringing rare disease therapy for patients in India

*AstraZeneca completed the acquisition of Alexion in the year 2021 and is now expanding its portfolio globally to bring innovative therapies for patients living with rare diseases in India

*Receives regulatory approval for its molecule ‘Selumetinib’ in India

Mumbai, India – 28 February 2022: AstraZeneca India, a science-led biopharmaceutical company today announced its entry into rare disease therapy in India. The regulatory approval of its ‘Selumetinib’ demonstrates company’s commitment to bring first in class, science-based therapies to patients in India. Through the addition, the company aims to address the severe unmet need of patients living with rare diseases and transform their quality of life.

In India, there is neither a clear estimate of the rate of mortality or morbidity nor an understanding of the disease burden on public healthcare system, when it comes to rare diseases. However, it is said that more than 400 million of population globally is affected by rare diseases and there are about 7000 recorded and identified rare diseases currently. It implies that there is a need to collectively further access as well as research in rare diseases so that we are better prepared to develop right medicines for patients diagnosed or living with known rare diseases and fight the burden.

“Patients with rare diseases need early and accurate diagnosis, timely treatment, long term supportive care. For this, government support is as essential as philanthropic or private support. Awareness among all stakeholders like the general public, medical fraternity, basic and clinical researchers, and policy makers is important. With less than 6% rare diseases having any kind treatment, coupled to extremely limited availability of any rare disease treatment in India, expectations from global pharma companies are high. New products launch by companies like AstraZeneca is important for rare disease patients, especially the neurological group of patients and they too can hope to live longer like any other population” said Prasanna Shirol, Co-founder and Executive Director, ORDI (Organization for Rare Diseases India).

“As a country, we need to accomplish a lot in this therapy area-right from diagnosis, treatment, access and education. With this milestone approval, we are bringing transformative medicines to patients living with rare diseases in India who currently have limited treatment options or no available treatment at all. By understanding patients’ unique needs, we can research and develop innovative medicines, support access and advocate for the rare disease community. We look forward to advancing this commitment and working with stakeholders to not only bring more innovative therapies for even more rare disease patients but also develop a roadmap to augment early diagnosis and holistic care”, said Dr Sanjeev Panchal, Country President and Managing Director, AstraZeneca India.

This rare disease day, AstraZeneca is also joining hands with Organization of Rare Diseases in India (ORDI), a non-profit organization working to improve the life of patients living with rare diseases. The collaboration is primarily to showcase support behind the cause and enhance the conversation. As a gesture of its support, AstraZeneca will be participating in ORDI’s flagship ‘RaceFor7’, a multi-city 7 km run commemorating Rare Diseases day in the country.

Being a science-led patient centric organization, AstraZeneca is focused on understanding patients’ needs and working with the ecosystem to improves awareness about rare diseases. The landscape of rare diseases is constantly evolving with new diseases identified from time to time but due to lack of awareness amongst medical community and general public, currently it takes 7 years on an average to diagnose a rare disease correctly. New molecular techniques and DNA sequencing technology have contributed significantly since it is found that almost 80% of all rare diseases have a genetic etiology. The new collaboration between AstraZeneca and ORDI aspires to leverage the latest science to support rare disease patients and their families in India.

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