China Net Finance, November 7th (Reporter Jia Yujing) - Recently, the world's leading innovative pharmaceutical and health enterprise Sanofi VI went to the China International Fair and unveiled several similar groundbreaking products in the innovation pipeline and immune fields of the group. Among them, the world's first siRNA therapy applied in the field of hemophilia is the innovative drug Fitusiran, and the latest approved new generation enzyme replacement therapy (ERT) drug for the treatment of Pompeii disease is resistant and praised; Reg; It is particularly worth mentioning.
It is reported that hemophilia is a hereditary hemorrhagic disease that lasts for a lifetime. Patients are referred to as "glassy people", and due to a natural lack of specific coagulation factors in the body, they suffer from "continuous blood flow" throughout their lives. As the largest disease among rare diseases, the incidence of hemophilia in China is approximately (2.73-3.09) per 100000.
Currently, the standard treatment for hemophilia is to receive 2-3 prophylactic intravenous injections of coagulation factors per week. However, patients still face high infusion frequency, poor treatment compliance, inability to ensure continuous and sufficient preventive treatment, and high disability rates, resulting in huge unmet needs. Sanofi is developing a new drug, Fitusiran, which has brought a breakthrough prevention and treatment plan for hemophilia A/B patients. It is reported that Fitusiran is the world's first and only siRNA hemophilia treatment drug, with injection frequency reduced by more than 90% compared to standard factor prophylactic treatment.
Sun Jing, a professor of hematology at Southern Hospital of Southern Medical University, stated that Fitusiran is a preventive treatment plan available for all types of hemophilia patients, and is effective for both type A and type B hemophilia patients with or without inhibitors. By reducing the level of antithrombin III, the annual bleeding rate of patients can be reduced by up to 90%.
Compared to the current routine treatment method of intravenous injection 3-4 times a week, Fitusiran only needs to undergo a minimum of 6 subcutaneous injections per year, greatly reducing the treatment burden on patients. As a healthcare worker, Sun Jing is pleased to see the upgrading and iteration of hemophilia treatment technology. In her opinion, preventive treatment is more effective in preventing disability than on-demand treatment.
Guan Tao, the founder and chairman of the Blood Friends Home and the chairman of the Beijing Blood Friends Home Rare Disease Care Center, stated that the treatment of hemophilia in China, especially in terms of medication, has undergone a change from "immortality" to "no pain", and will continue to pursue "no disability". Adequate prevention and treatment is the only way to achieve 'no disability' for hemophilia. Fitusiran's convenient and easy to use administration has fundamentally changed patients' diagnosis and treatment habits, opening a new era for hemophilia prevention and treatment
In addition to developing a new drug, Fitusiran, Sanofi's new generation of enzyme replacement therapy (ERT) drugs for the treatment of Pompeii disease are highly resistant; Reg; We also took advantage of our efforts to complete the magnificent transformation from "exhibits" to "products" at the Expo. It is reported that Nai Er Zan& Reg; Last year, it made its debut in China and was approved in China on the eve of this year's promotion.
Public information shows that Pompeii's disease generally refers to glycogen storage disease type II. The neurological manifestations of this disease are mainly motor disorders, with muscle weakness in the limbs, muscular atrophy, pseudohypertrophy, and other muscle diseases being more common. According to Zhao Zhongbo, chief physician of the Department of Neurology at Huashan Hospital affiliated with Fudan University, Pompeii disease is an autosomal recessive genetic disease and an extremely rare disease with a very small number of patients. If left untreated, it can lead to irreversible and progressive damage to multiple organs and systems, requiring long-term dependence on wheelchairs and ventilators, and leading to premature death.
In October of this year, Sanofi's next-generation enzyme replacement therapy (ERT) drug resistance was praised; Reg; Approved by the National Drug Administration (NMPA) of China for long-term treatment of Pompeii disease patients. According to the introduction, Nai Er Zan& Reg; Awarded the title of "breakthrough therapy" by the FDA, its 15-fold enzyme transporter level can increase cell uptake of enzymes, better improve respiratory and motor function in patients with Pompeii disease, with good safety, and is aimed at becoming a new generation of standard treatment plan.
Professor Fu Lijun from the Shanghai Children's Medical Center affiliated with Shanghai Jiao Tong University School of Medicine stated that patients with Pompeii disease also look forward to more innovative drugs to bring them better quality of life benefits when receiving treatment and delaying disease progression. Speak of patience and praise& Reg; Approved in China, Fu Lijun stated that clinical data shows that the drug is highly recommended; Reg; It can be more efficiently targeted for uptake to clear glycogen, improve respiratory function and exercise ability of patients with Pompeii disease, and improve the quality of life of patients with Pompeii disease. I am glad to see the new generation of ERT drugs for Pompeii disease approved in China, and I hope to let the public know about Pompeii disease and its new therapies through the platform of the CIIE
Xie Lijuan, General Manager of Sanofit China, stated that with the approval of NIZAN in China, Chinese patients with Pompeii disease will receive a new revolutionary treatment and new hope. Nerozan has also expanded Sanofi's rare disease product portfolio in China - to date, it has covered lysosomal storage disorders such as Gaucher's disease, Pompeii's disease, Fabry's disease, and mucopolysaccharide storage type I, as well as neuroscience diseases such as multiple sclerosis and frostbite. In the future, Sanofi will continue to enhance its product line for rare diseases, introduce innovative and high-quality drugs to Chinese patients, and work together to build an ecosystem covering prevention and treatment, helping rare disease patients face health challenges and regain their lives
The CIIE is an open platform for innovation and cooperation, seeking opportunities together. Patients with rare diseases should also share their health with the general public in the new era. In addition to therapeutic innovation, breakthrough policy mechanism innovation is also needed to ensure drug accessibility and affordability, ultimately helping patients achieve sustained benefits. At this year's CIIE, Sanofi also assisted the Koude Rare Disease Center and several authoritative experts in the field of rare diseases to jointly launch the "China Rare Disease Legislative Protection Exploration Initiative Action", exploring the "Chinese model" of rare disease protection for the vast number of rare disease patients through product and institutional innovation.