AstraZeneca Launches Eculizumab in China for Treatment of Atypical Hemolytic Uremic Syndrome and PNH

　　【Pharmaceutical Network Product Information】 Due to the complexity of the environment, dietary structure within society, and other factors, the number of people with uremia continues to rise, showing a trend of becoming younger. Therefore, finding effective and critical prevention methods is an urgent task, and strengthening the development of related drugs is also imperative. The author learned that on November 11, another innovative drug was announced to be launched in China.
 

On November 11, AstraZeneca announced that the innovative rare disease drug eculizumab (brand name: Soliris), used to treat adults and children with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, has officially launched and will soon be available in cities such as Suzhou, Qingdao, Beijing, Shanghai, Tianjin, and Hangzhou.
 

Data show that eculizumab is a globally approved C5 complement inhibitor that works by selectively inhibiting the activation of the terminal complement C5 protein. It has been approved in markets such as the United States, the European Union, and Japan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG) in adult patients who are positive for anti-acetylcholine receptor (AChR) antibodies, and neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are positive for anti-aquaporin-4 (AQP4) antibodies.
 

It is reported that atypical hemolytic uremic syndrome is a continuous complement-mediated systemic thrombotic microangiopathy, which often presents acutely and has a much higher incidence in children than in adults. Chronic uncontrolled complement activation leads to complement-mediated thrombotic microangiopathy (CM-TMA), forming blood clots in small blood vessels throughout the body. Patients may face a lifelong risk of TMA, causing sudden, life-threatening damage to the kidneys and other vital organs.
 

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, life-threatening rare blood disorder that can occur at any age but is more common in young and middle-aged adults. Previously, the treatment options for PNH were mainly symptomatic, including blood transfusions, corticosteroids, and immunosuppression, yet many challenges remained in controlling hemolysis and preventing renal damage.
 

Eculizumab was approved in China in 2018 and passed the application for the change of the new production site of the original solution this August. It is used to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome in adults and children. The drug's market entry also brings benefits to patients, meeting new medication needs.
 

AstraZeneca stated that rare diseases are a significant health challenge faced globally. In the future, AstraZeneca will continue to support various partners in exploring multi-level security models, helping to reduce the medical burden on patients with rare diseases and enhance their healthcare security rights.
 

Moreover, China's attention to the rare disease population is increasing, and significant progress has been made in multiple fields such as rare disease research, diagnosis and treatment, drug development and supply, and the medical insurance system. In China, uremia is highly prevalent, and even many young people are deeply troubled by it; therefore, it is urgent to strengthen drug innovation and research in this field.
 

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