AstraZeneca India expands its portfolio with rare disease therapy

AstraZeneca India has declared its entry into the Indian market for rare disease therapy. The regulatory approval of its 'Selumetinib' shows the company's commitment to bringing first-in-class, science-based therapies to patients in India. With this addition, the business hopes to address a critical unmet need for patients living with rare diseases and improve their quality of life. When it comes to rare diseases, India lacks a clear estimate of mortality or morbidity rates, as well as a knowledge of the disease burden on the public healthcare system.

However, it is estimated that more than 400 million people worldwide are affected by rare diseases, and there are presently approximately 7000 recorded and identified rare diseases. It implies that there is a need for us to collectively advance access and research in rare diseases so that we are better equipped to create the appropriate medicines for patients diagnosed or living with known rare diseases and fight the burden.

"Patients with rare illnesses require an early and precise diagnosis, prompt treatment, and long-term supportive care. Government assistance is just as important as charitable or private assistance in this regard. It is critical to raise awareness among all stakeholders, including the general population, the medical community, basic and clinical researchers, and policymakers. With less than 6% of rare diseases having any kind of treatment, and highly limited availability of any rare disease treatment in India, global pharma firms have high expectations. "New products introduced by companies like AstraZeneca are essential for rare disease patients, particularly the neurological group, and they, like any other population, can expect to live longer," said Prasanna Shirol, Co-founder and Executive Director, ORDI (Organisation for Rare Diseases India).

"As a country, we need to accomplish a lot in this therapy field, straight from diagnosis, treatment, access and education. With this landmark approval, we are bringing transformative medicines to patients in India suffering from rare diseases who currently have few or no treatment choices. By understanding patients' unique requirements, we can study and create innovative medicines, support access and advocate for the rare disease community. We look forward to advancing this commitment and collaborating with stakeholders to not only bring more innovative therapies to even more rare disease patients, but also to develop a roadmap to supplement 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 the Organisation of Rare Diseases in India (ORDI), a non-profit organisation working to improve the life of patients living with rare diseases. The collaboration primarily showcases support behind the cause and enhances the conversation. The landscape of rare diseases is constantly evolving with new diseases identified from time to time but due to a lack of awareness amongst the medical community and general public, currently, it takes seven years on average to diagnose a rare disease correctly. New molecular techniques and DNA sequencing technology have contributed significantly since it is found that almost 80 per cent of all rare diseases have a genetic aetiology. The new collaboration between AstraZeneca and ORDI aspires to leverage the latest science to support rare disease patients and their families in India.