Takeda's Innovative Drug Obizur (Susoctocog alfa) Receives CDE Acceptance for NDA in China for Acquired Hemophilia A

ShanghaiJune 16, 2022PR Newswire -- Takeda China announced today that the New Drug Application (NDA) for its innovative drug Recombinant Antihemophilic Factor (Porcine Sequence) Obizur (Susoctocog alfa) has been officially accepted by the Center for Drug Evaluation of the National Medical Products Administration. It is intended for on-demand treatment and control of bleeding episodes in adult patients with Acquired Hemophilia A (AHA). Obizur (Susoctocog alfa) is currentlyThe FirstU.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA)^[1]，[2]Recombinant porcine FⅧ (rpFⅧ) approved for acquired hemophilia A is expected to provide a new treatment option for patients with acquired hemophilia A in China.

Acquired Hemophilia A is an acquired bleeding disorder. Patients experience reduced factor VIII (FⅧ) activity due to the presence of autoantibodies against coagulation factor VIII in circulating blood. The condition is characterized by spontaneous bleeding in individuals with no prior history of bleeding or positive family history, or abnormal bleeding during surgery, trauma, or invasive procedures. The annual incidence rate is approximately 1.5 per million.^[3]. The disease is characterized by its rarity and sudden onset, with heterogeneous bleeding manifestations ranging from no bleeding tendency or only mild bleeding to potentially fatal events such as gastrointestinal bleeding, retroperitoneal bleeding, and intracranial hemorrhage. The China Acquired Hemophilia Registry (CARE) study showed that severe bleeding occurred in approximately 60.9% of patients with acquired hemophilia A at their first medical visit.^[4], posing a serious threat to life and health. Due to the dangerous nature of acquired hemophilia A, timely diagnosis and early administration of appropriate treatment are key to the successful treatment of acquired hemophilia A.^[5]。

Obizur (Susoctocog alfa) is structurally similar and sequence homologous to human coagulation factor VIII (FVIII), and is not easily inactivated by anti-human FVIII autoantibodies, allowing it to replace human FVIII in exerting significant hemostatic effects. In a prospective, phase 2/3, multicenter, open-label clinical trial, a total of 28 patients with acquired hemophilia A were enrolled, and 100% of the patients showed an evaluable response (bleeding stopped or decreased) within 24 hours after administration. Among them, 94% (16/17) of the patients received rpFVIII as first-line treatment. Additionally, as a recombinant protein, Obizur reduces the risk of transmission of blood-borne pathogens.^[6]。

Takeda has over seventy years of heritage in the field of rare blood disease treatment. It has long been committed to innovative research and development in this area, boasting a wide portfolio of up to 11 products covering the treatment of various rare blood diseases. In China, Takeda has steadfastly upheld its commitment to "putting patients first" over the years, working with all parties to promote the construction of a rare blood disease diagnosis and treatment system, popularizing standardized preventive treatment concepts, and striving to improve the living conditions and treatment status of Chinese blood disease patient groups, including hemophilia patients, contributing tirelessly towards achieving the goal of "Healthy China" for all citizens. Since 2013, Takeda has successively introduced advanced prophylactic treatments and personalized treatment solutions in the hemophilia field, and will accelerate the introduction of more innovative drugs for rare blood diseases into the Chinese market in the coming years, covering acquired hemophilia, von Willebrand disease, thrombotic thrombocytopenic purpura, etc., to further meet the unmet needs of Chinese patients with rare blood diseases.

Statement

1. This article aims to convey cutting-edge pharmaceutical information and does not constitute a recommendation or promotion of any drug or treatment plan.

2. If you want to learn more about disease knowledge or drug and treatment-related information, please consult a healthcare professional.

3. This article is provided by Takeda China for media reference only, and the final published version shall be determined by each media outlet.

[1] https://investor.baxter.com/investors/events-and-news/news/press-release-details/2014/FDA-Approves-Baxters-OBIZUR-Antihemophilic-Factor-Recombinant-Porcine-Sequence-for-Acquired-Hemophilia-A/default.aspx.

[2] EU Approves Obizur for the Treatment of Adults with Acquired Hemophilia [J]. Journal of Clinical Rational Drug Use, 2015(36):1.

[3] Thrombosis and Hemostasis Group, Hematology Branch of Chinese Medical Association, et al. Chinese Journal of Hematology, 2021, 42(10): 793-799.

[4] SunB, XueF, FengY, et al. Outcome of CARE: a 6-year national registry of acquired haemophilia A in China[J]. Br J Haematol, 2019, 187(5): 653-665. DOI: 10.1111/bjh.16128.

[5] Chinese Guidelines for the Diagnosis and Treatment of Acquired Hemophilia A (2021 Edition)

[6] Haemophilia(2015),21, 162-170