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CME Evaluation

Oral Communication Session

Inherited Bleeding Disorders and Inhibitors

Session: Novel hemophilia treatment, clinical trials I

OC 40.1 - A Novel Extended half-life factor VIII Fc fusion protein FRSW107 for severe hemophilia A: A multicentre, open-label, single-arm, phase 3 study and its open-label extension

Monday, June 24, 2024
14:45 – 15:00 ICT
Room: Plenary Hall

    Presenting Author(s)

  • Feng Xue, MD

    Chief physician
    State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin 300020, China
    Tianjin, Tianjin, China (People's Republic)

    • Co-Author(s)

  • Hu Zhou, MD

    Chief Physician
    Department of Hematology, The Affiliated Cancer Hospital of Zhengzhou University & Henan Cancer Hospital
    Zhengzhou, Henan, China (People's Republic)

  • YC

    Yun Chen

    professor
    Jinan central hospital, Shandong, P.R. China
    Jinan, Shandong, China (People's Republic)

  • Jing Sun, MD

    No. 1838 North Guangzhou Avenue, Guangzhou, China
    Department of Hematology, Nanfang Hospital, Southern Medical University
    Guangzhou, Guangdong, China (People's Republic)

  • CJ

    Chenghao Jin

    professor
    Jiangxi Provincial People's Hospital, Jiangxi, P.R. China
    Jiangxi, Jiangxi, China (People's Republic)

  • PL

    Pingchong Lei

    professor
    Henan provincial People's Hospital, Henan, P.R. China
    zhenzhou, Henan, China (People's Republic)

  • ZL

    Zhenyu Li

    professor
    The Affiliated Hospital of Xuzhou Medical College, Jiangsu, P.R. China
    Xuzhou, Jiangsu, China (People's Republic)

  • YX

    Yaming Xi

    professor
    The First Hospital of Lanzhou University, Gansu, P.R. China
    Lanzhou, Gansu, China (People's Republic)

  • XZ

    Xiaojing Zeng

    professor
    The Affiliated Hospital of Guizhou Medical University, Guizhou, P.R. China
    Guizhou, Jiangxi, China (People's Republic)

  • ZC

    Zhongguang Cui

    professor
    The Affiliated Hospital of Qingdao University, Shandong, P.R. China
    Qingdao, Shandong, China (People's Republic)

  • XZ

    Xielan Zhao

    professor
    Xiangya Hospital of Central South University, Hunan, P.R. China
    changsha, Hunan, China (People's Republic)

  • LY

    Linhua Yang

    professor
    The Second Hospital of Shanxi Medical University, Shanxi, P.R. China
    Shanxi, Shanxi, China (People's Republic)

  • YY

    Yi Yang

    professor
    Chongqing University Three Gorges Hospital, Chongqing, P.R. China
    Chongqing, Chongqing, China (People's Republic)

  • BT

    Baolin Tang

    Doctor
    Anhui Provincial Hospital, Anhui, P.R. China
    Hefei, Anhui, China (People's Republic)

  • AS

    Aizong Shen

    professor
    Anhui Provincial Hospital, Anhui, P.R. China
    Hefei, Anhui, China (People's Republic)

  • Runhui Wu, PhD, MD

    Professor
    Department of Hematology, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, 100045, China
    Beijing, Beijing, China (People's Republic)

  • XW

    Xiaolin Wang

    professor
    Capital Medical University affiliated Beijing Children's Hospital, Beijing, P.R. China
    Beijing, Beijing, China (People's Republic)

  • LL

    Ling Liu

    professor
    The Second Affiliated Hospital of Guangzhou Medical University, Guangzhou, P.R. China
    Guangzhou, Guangdong, China (People's Republic)

  • Jl

    Jiwei lou

    professor
    Gensciences Inc.,Nantong, P.R. China
    Nantong, Jiangsu, China (People's Republic)

  • Renchi Yang, MD

    Head of Thrombosis and Hemostasis Center
    Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
    Tianjin, Hebei, China (People's Republic)

Background: Hemophilia A is a rare hereditary disease caused by a deficiency of coagulation factor VIII (FVIII).

Aims: This multicentre, open-label, single-arm phase 3 trial and its open-label extension aimed to assess the efficacy and safety of a novel extended half-life factor VIII (FVIII) Fc fusion protein FRSW107 as prophylactic and on-demand treatment for severe hemophilia A.

Methods: Between October 9, 2020 and June 26, 2022, adolescents and adults with severe haemophilia A (FVIII activities < 1 IU/dL) without FVIII inhibitors received intravenously FRSW107 50 IU/kg Q3D for 50 exposure days and at least six months for prophylaxis or FRSW107 30 to 50 IU/kg for six months for on-demand therapy. The primary outcomes were the annualized bleeding rate (ABR), annualized joint bleeding rate (AJBR) and number of target joints.

Results: Eighty-three patients received prophylaxis and 36 received on-demand treatment; 101 entered the extension period. FRSW107 had a mean elimination half-life of 20.1±4.7 h and a mean incremental recovery of 2.1±0.5 IU/dL/IU/kg. By exposure day 100, 53 (63.9%) patients in the prophylaxis group had zero bleed. The mean ABR was 1.5 ± 3.8 events (95% CI, 1.0-2.3), with a 95.3% reduction from baseline (p < 0.0001). The mean AJBR was 1.2±3.5 events (95% CI, 0.8-1.9), with a 95.8% reduction from baseline (p < 0.0001). The mean number of target joints was 0.1±0.3 (95% CI, 0.0-0.1), representing a 96.9% reduction from baseline (p < 0.0001). Treatment-related adverse events occurred in 19 (16.0%) patients but caused no treatment interruption, discontinuation, withdrawal or death.

Conclusion(s): FRSW107 was well tolerated and efficacious in the prophylactic and episodic treatment of bleeding events in previously treated adolescents and adults with severe hemophilia A.