China Approves First Long-Acting Recombinant Factor IX Therapy Alprolix® to Reduce Disability Rates in Pediatric Hemophilia B Patients

SHANGHAI, April 23, 2021 /PRNewswire/ -- Sanofi announced today that the China National Medical Products Administration has approved Eloctate (recombinant human coagulation factor IX-Fc fusion protein for injection; brand name: Alprolix)^®, English brand name Alprolix^®) for the control of bleeding, routine prophylaxis, and perioperative bleeding management in patients with confirmed hemophilia B (congenital factor IX deficiency) in adults and children. AsThe World's FirstRecombinant Human Coagulation Factor IX Fc Fusion Protein (rFIXFc), Alprolix^®Significantly Prolongs the Half-life of Coagulation Factors and Reduces Injection Frequency in Patients^{[1], [2]}, particularly suitable for prophylactic treatment in children, promoting the recovery of target joints, and providing comprehensive and sustained hemostatic protection for patients with hemophilia.

Urgent Clinical Need: Reducing Injection Frequency to Achieve Long-Term Protection 

Hemophilia is a rare recessive hereditary bleeding disorder. There are approximately 38,000 patients with hemophilia in China, among whom 15%–20% have hemophilia B.^[1]. Patients with hemophilia B face a lifelong risk of severe bleeding due to deficient or low activity of coagulation factor IX in their bodies. In the past, maintaining a certain level of coagulation factor IX in patients with hemophilia B required daily infusions of factor IX.^[3]. This poses significant challenges to patient adherence and prognosis; therefore, there is an urgent clinical need for therapeutic approaches that reduce injection frequency and improve patient adherence.

Sanofi^®Fc fusion technology extends the plasma half-life and duration of efficacy of Factor IX, significantly reducing dosing frequency. In routine prophylactic therapy, the injection frequency of clotting factors, previously several times per week, has been reduced to an average of once weekly.^[4]. In May 2019, Sanofi^®Listed in the second batch of overseas new drugs urgently needed for clinical use by the Center for Drug Evaluation, National Medical Products Administration.

Professor Yang Renchi, Director of the Thrombosis and Hemostasis Diagnosis and Treatment Center at the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, pointed out, “Saijiuning^®Its approval has introduced a long-acting hemostatic regimen for patients with hemophilia B in China. While significantly reducing the annualized bleeding rate (ABR), it also decreases the frequency of injections required by patients. Furthermore, its annual factor consumption is half that of non-long-acting recombinant factors currently available on the market, thereby further conserving medical resources and alleviating the disease burden on patients and their families caused by frequent injections.

Two multicenter, open-label Phase III clinical studies (B-LONG and Kids B-LONG) and one extension study (B-YOND) demonstrated that in terms of bleed prevention, switching from traditional recombinant Factor IX (rFIX) prophylaxis administered once weekly to Alprolix^®Among patients receiving prophylactic treatment, the annualized bleeding rate (ABR) decreased from 5.5 to 2.9 with individualized prophylaxis using Sevocog alfa^®Can reduce ABR by 80% or more^{[5], [6], [7]}, with over one-third using Sanofi's products^®Patients Receiving Individualized Prophylactic Therapy Achieve Zero Spontaneous Bleeding Episodes^{[5], [8]}. In terms of controlling bleeding, data show that in 80.8% of major surgeries, only a single injection of Saijiuning was required^®Hemostasis can be maintained.^[9]。

Childhood Prophylactic Treatment Effectively Reduces Disability Rates

More than 90% of bleeding episodes in patients with hemophilia occur in the joints.^[10], often with onset in childhood^[11]Recurrent, long-term joint bleeding can not only lead to deformity and disability but may even be life-threatening. Therefore, prophylactic treatment during childhood is particularly important for patients with hemophilia. The Kids B-LONG study showed: Eloctate^®In prophylactic treatment, the annualized spontaneous bleeding rate (AsBR), including at joint sites, was zero in pediatric patients across all age groups.^[12]。

Professor Wu Runhui, Chief Physician at the Hematology and Oncology Center of Beijing Children’s Hospital Affiliated to Capital Medical University, believes that “Sa Jiu Ning^®Its approval has enriched the prophylactic treatment options for patients with hemophilia B, helping to slow the progression of arthropathy and improve patients’ quality of life.^[13]“For pediatric patients with hemophilia, the benefits are even more pronounced, as childhood represents the golden period for hemophilia management; initiating standard prophylactic therapy during this time yields enhanced efficacy with reduced effort, effectively lowering the rate of disability.”

Dr. Henriette He, President of Sanofi Greater China, stated: “As a global leader in the field of rare diseases, Sanofi is proud to expand our innovative product portfolio in China to include rare hematologic disorders. Saijiuning^®The approval will bring new treatment options for children with hemophilia, addressing unmet clinical needs. Prophylactic treatment during childhood is critical in hemophilia management. We will actively collaborate with all stakeholders to accelerate the availability of Sevacix^®"Launch on the market as soon as possible to benefit patients."

[1] Rath et al. Crit Rev Biotechnol 2015;35(2):235-54. (p. 240)
[2] Bleeker et al. Blood 2001;98:3136-3142. (p. 3136-37).
[3] Thrombosis and Hemostasis Group, Hematology Branch of Chinese Medical Association; Chinese Hemophilia Collaboration Group. Chinese expert consensus on the diagnosis and treatment of hemophilia (2017 edition) [J]. Chinese Journal of Hematology, 2017, 38(5): 364-370. DOI: 10.3760/cma.j.issn.0253-2727.2017.05.002.
[4] Buckley BC et al. Real-World Dosing and Patient Characteristics of rFIXFc in Hemophilia B Patients. Poster presented at the Academy of Managed Care Pharmacy Nexus Meeting. Orlando, FL, USA; Oct 26-29, 2015.
[5] Powell JS et al. N Engl J Med 2013;369:2313-23.
[6] Pasi KJ et al. Thromb Haemost 2017;117(3):508-18.
[7] Ragni M et al. 2018. Abstract submitted to the 70th Annual Meeting of the National Hemophilia Foundation. Orlando, FL, USA; Oct 11-13, 2018.
[8] Fischer K et al. Lancet Haematol 2017;4(2):e75-82.
[9] ALPROLIX Prescribing Information. 2018 Waltham, MA, USA. Bioverativ Therapeutics Inc.
[10] Simpson ML and Valentino LA. Expert Rev Hematol 2012;5(4):459-68.
[11] Guidelines for the Diagnosis and Treatment of Hemophilia in Children
[12] Fischer K, et al. Lancet Haematol. 2017 Feb;4(2):e75-e82.
[13] Srivastava A, et al. Haemophilia 2013;19(1):e1-47.