AstraZeneca China's first rare disease drug Sulirui achieved milestone progress in China, providing new options for rare disease treatment

The China National Medical Products Administration approved the application for the change of the new origin of Eculizumab injection (trade name: Soliris®, Soliris®)

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, and life-threatening rare hematologic disorder characterized by complement-mediated intravascular hemolysis, underlying hematopoietic failure, and thrombotic tendencies, resulting in Organ damage or even death

Atypical hemolytic uremic syndrome (aHUS) is a complement-mediated thrombotic microangiopathy (CM-TMA) caused by an overreaction of the body's complement system to attack healthy cells

As an approved C5 complement inhibitor, eculizumab works by selectively inhibiting the activation of the terminal complement C5 protein

Professor Wu Depei, director of the Hematology Department of the First Affiliated Hospital of Soochow University, executive deputy director of the National Clinical Research Center for Hematological Diseases, and chairman of the Chinese Medical Association Hematology Branch, said: "In recent years, PNH has made new progress in diagnosis and treatment, and Ikuzhu Monoclonal antibodies provide a new option for controlling hemolysis and achieving long-term survival of patients

Professor Zhao Minghui, director of the Institute of Nephrology at Peking University and vice chairman of the Chinese Medical Association Nephrology Branch, said: "aHUS patients continue to be at risk of systemic, life-threatening and sudden complications

       Wang Lei, global executive vice president of AstraZeneca, president of international business and China, said: "In order to drive the development of global rare disease medicine, AstraZeneca has fully integrated the advantageous resources of Rayson Pharma in the field of rare diseases.

       In 2021, AstraZeneca entered the field of rare diseases with the acquisition of Ruisong Pharmaceuticals, and in September of the same year, the Rare Diseases Division was established in China
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At present, AstraZeneca’s rare disease research and development has been deployed in six major disease areas: blood, kidney, central nervous system, metabolism, cardiovascular, and ophthalmology
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In the future, a variety of new drugs will be introduced, involving complement and non-complement systems, and will be gradually developed simultaneously with the world
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In response to the country's call for innovative drugs to be tested first, AstraZeneca and the Hainan Boao Lecheng International Medical Tourism Pilot Area Administration signed the "Strategic Cooperation Agreement for the First and First Test of Innovative Drugs for Rare Diseases" to jointly promote the early use and innovation of innovative drugs for rare diseases Drug introduction and other projects
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In June 2022, AstraZeneca and the Qingdao Municipal Government formally signed a memorandum of cooperation, planning to set up a rare disease-themed innovation center, a life science innovation park and an industrial fund in Qingdao, linking high-quality resources at home and abroad, and jointly building a broad influence The ecological highland of rare disease innovation
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       Disclaimer: The other indications involved in this article have not been approved in mainland China, and AstraZeneca does not recommend the use of any unapproved drugs
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