AstraZeneca India, a science-led biopharmaceutical company 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.