AstraZeneca India, a biopharmaceutical company, on Monday, announced its entry into rare disease therapy in India after receiving the regulatory approval for ‘Selumetinib’.

It aims to address the severe unmet needs 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 the public healthcare system, when it comes to rare diseases. According to the company, more than 400 million people worldwide are affected by rare diseases, and currently, there are about 7000 recorded and identified rare diseases.

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“Patients with rare diseases need an early and accurate diagnosis, timely treatment, and long-term supportive care. With less than 6 per cent of rare diseases having any kind of treatment, coupled with the extremely limited availability of any rare disease treatment in the country, expectations from global pharma companies are high,” said Prasanna Shirol, Co-founder and Executive Director of ORDI (Organisation for Rare Diseases India).

Sanjeev Panchal, Country President and Managing Director, AstraZeneca 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.”

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On February 28, 2023—the day dedicated to rare diseases—AstraZeneca also joined hands withORDI, a non-profit organisation working to improve the lives of patients living with rare diseases.

The collaboration is primarily to showcase support for 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-kilometer run commemorating Rare Disease Day in the country.