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【Pharmaceutical Network Product Information】 Due to the complexity of the environment, the diet structure in the society and other factors, the number of uremia is rising, and it is showing a younger trend, so it is urgent to find effective and key prevention methods, and it is urgent to strengthen the development of related drugs
.
The author learned that on November 11, another innovative drug was announced to be launched
in China.
That is, on November 11, AstraZeneca announced that the rare disease innovative drug eculizumab (trade name: Sulirui), a rare disease innovative drug for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome in adults and children, was officially launched and will land
in Suzhou, Qingdao, Beijing, Shanghai, Tianjin, Hangzhou and other cities as soon as possible 。 According to the data, eculizumab is a globally approved C5 complement inhibitor that works by selectively inhibiting the activation of terminal complement C5 protein, and is approved by the United States, the European Union and Japan for the treatment of PNH, atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) anti-positive systemic myasthenia gravis (gMG) adult patients and anti-aquaporin-4 (AQP4) anti-antibody positive adult
patients with neuromyelitis optica spectrum disorder 。 Atypical hemolytic uremic syndrome is a persistent complement-mediated systemic thrombotic microangiopathy with a more acute onset and a much higher incidence in children than in adults
.
Chronic uncontrolled activation of complement, leading to complement-mediated thrombotic microangiopathy (CM-TMA), which forms thrombus
in small blood vessels throughout the body.
Patients may be at 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 disease of the blood system that can occur at any age and tend to occur in young adults
.
Previously, the treatment of PNH was mainly symptomatic treatment, including blood transfusion, glucocorticoids, immunosuppression, etc.
, but it faced many problems
in hemolytic control and prevention of renal function damage.
Eculizumab was approved in China in 2018 and was approved in August this year through the application for change of origin of the original solution for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome
in adults and children.
The launch of the drug has also brought benefits to patients and provided new drug needs
.
AstraZeneca said that rare diseases are a major health issue facing the world, and in the future, AstraZeneca will continue to support partners to explore multi-level protection models to help reduce the medical burden of patients with rare diseases and improve medical security rights
.
In addition, China has paid increasing attention to rare disease groups, and many undertakings such as rare disease research, diagnosis and treatment, drug R&D and supply, and medical security system have made great
progress.
The high incidence of uremia in China, even many young people are deeply troubled, so it is urgent
to strengthen drug innovation and research in this field.
Disclaimer: Under no circumstances does the information or opinions expressed herein constitute investment advice
to anyone.