AstraZeneca's First Rare Disease Drug Soliris Achieves Milestone in China with Approval of New Drug Substance Site, Offering New Treatment Options for Patients

ShanghaiAugust 30, 2022PR Newswire -- Today, the National Medical Products Administration of China approved Eculizumab Injection (brand name: Soliris).^®，Soliris^®) to pass the application for the change of the new production site of the bulk solution. As a prescription drug approved in China for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in adults and children.^[1], this approval marks eculizumab as AstraZeneca's first rare disease drug in the Chinese market, taking a crucial step towards filling the gap in this field in China and providing Chinese patients in related rare disease areas with a new treatment option.

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a chronic, progressive, life-threatening rare blood disorder characterized by complement-mediated intravascular hemolysis, potential bone marrow failure, and a propensity for thrombosis, leading to organ damage and even death. The global prevalence of PNH is approximately 16-20 per 100,000.^[2], The current estimate is that the incidence rate in China is around 1/100,000.^[3]. The disease predominantly affects young adults, with 77% of PNH patients developing the condition between the ages of 20 and 40. Without effective treatment, PNH patients experience poor quality of life and shortened survival, with a five-year mortality rate of 35%.^[4]。

Atypical Hemolytic Uremic Syndrome (aHUS) is a complement-mediated thrombotic microangiopathy (CM-TMA) caused by the overreaction of the body's complement system attacking healthy cells. Patients with this extremely rare disease develop blood clots in small blood vessels throughout the body, leading to sudden life-threatening damage to the kidneys and other vital organs. The prevalence of aHUS is approximately 7 per million.³, with a sudden onset, and the incidence rate in children is higher than that in adults, with approximately 25% of children dying during the acute phase.^[5]Nearly 50% of aHUS patients can progress to end-stage renal disease (ESRD), with a mortality rate of 25%.^[6]。

As the world's first approved C5 complement inhibitor, eculizumab works by selectively inhibiting the activation of the terminal complement C5 protein. Currently, eculizumab has been approved for multiple indications in many countries and regions worldwide.^[7]A 26-week randomized, double-blind, placebo-controlled TRIUMPH trial evaluated the safety and efficacy of eculizumab in patients with PNH experiencing hemolysis. The results showed that, compared to the placebo group, patients treated with eculizumab had significantly improved hemolysis and reduced transfusion requirements.^[8]In addition, two key studies, C08-002 and C08-003, also confirmed the safety and efficacy of eculizumab in the treatment of aHUS. The results of the C08-002 study showed that over 80% of patients had their platelet counts return to normal and remain stable after receiving eculizumab treatment, and 88% of patients achieved and maintained a TMA-free status. In the C08-003 study, after 26 weeks of eculizumab treatment, 80% of patients reached a TMA-free state, and renal function also improved.^[9]To date, global data show that long-term safety and tolerability of eculizumab are good.^[10],[11]。

Professor Depei Wu, Director of the Hematology Department at the First Affiliated Hospital of Soochow University, Executive Vice Director of the National Clinical Research Center for Hematologic Diseases, and Chairman of the Hematology Society of the Chinese Medical Association, stated: "In recent years, new progress has been made in the diagnosis and treatment of PNH. Eculizumab provides a new option for controlling hemolysis and achieving long-term patient survival. At the same time, we are also highly anticipating the establishment of the China PNH Collaboration Network, which will be significant in improving the standardized treatment of this disease in China."

Professor Minghui Zhao, Director of the Peking University Institute of Nephrology and Vice Chairman of the Nephrology Society of the Chinese Medical Association, stated: "Patients with aHUS are at ongoing risk of systemic, life-threatening, and sudden complications. As the only C5 complement inhibitor approved in China for the treatment of aHUS, we highly anticipate the launch of eculizumab to help improve patient outcomes and enhance their quality of life."

Leo Wang, Executive Vice President of AstraZeneca Global, President of International Business and China, stated: "To drive the development of global rare disease medicine, AstraZeneca has fully integrated the advantageous resources of Alexion Pharmaceuticals in the rare disease field. With its leading scientific innovation capabilities and forward-looking layout in the rare disease area, AstraZeneca is making every effort to accelerate the introduction of innovative global rare disease drugs into China while actively promoting the localization of rare disease diagnosis and treatment in China. We are very pleased to witness this milestone for eculizumab, which also marks that AstraZeneca will soon welcome its first rare disease drug in China. In the future, we will continue to collaborate with local governments and partners to improve the accessibility of innovative rare disease drugs, support the establishment of a sound rare disease diagnosis and treatment ecosystem, and jointly create a future where rare diseases are no longer rarely treated."

In 2021, AstraZeneca entered the rare disease field by acquiring Alexion Pharmaceuticals and established a Rare Disease Business Unit in China in September of the same year. Currently, AstraZeneca's rare disease research and development has been deployed across six major disease areas: hematology, nephrology, central nervous system, metabolism, cardiovascular, and ophthalmology. In the future, multiple new drugs involving both complement and non-complement systems will be introduced, gradually achieving synchronized R&D with the rest of the world. In response to the national call for the early adoption and pilot use of innovative drugs, AstraZeneca signed a "Strategic Cooperation Agreement for Early Adoption and Introduction of Innovative Rare Disease Drugs" with the Administration of Boao Lecheng International Medical Tourism Pilot Zone in Hainan, jointly promoting projects such as early access to innovative rare disease drugs. In June 2022, AstraZeneca formally signed a Memorandum of Cooperation with the Qingdao Municipal Government, planning to establish an innovation center, life science park, and industrial fund focused on rare diseases in Qingdao, linking high-quality resources from home and abroad, and jointly building a rare disease innovation ecosystem with extensive influence.

Disclaimer: The other indications mentioned in this article have not been approved in China, and AstraZeneca does not recommend the use of any unapproved drugs.