Takeda's PK-Guided Hemophilia Management Tool myPKFiT® Approved in China, Ushering in a New Era of Personalized Treatment for Patients with Hemophilia A

Takeda China Announces Today a Pharmacokinetics (PK)-Guided Management Tool for Hemophilia A (Congenital Factor VIII Deficiency)myPKFiT^®Approved by the National Medical Products Administration (NMPA),Indicated for patients aged 16 years and older (weighing 45 kg or more) receiving Advate^®patients with hemophilia A treated with (recombinant human coagulation factor VIII for injection).myPKFiT^®It is China’s first management tool supporting hemophilia pharmacokinetics (PK)-guided care, and to date, the only individualized prophylactic FVIII dosing guidance software approved by the National Medical Products Administration.For use by licensed healthcare professionals (HCPs) familiar with the treatment of hemophilia A. Baiyin Zhi^®with myPKFiT^®When used in combination, it enables personalized adjustment of Factor VIII (FVIII) dosing for hemophilia patients based on their individual characteristics and needs, ushering in a new era of personalized treatment for hemophilia patients in China and empowering them to live life with greater freedom.

Hemophilia is a rare recessive genetic disorder primarily characterized by coagulation impairments due to a deficiency in clotting factors. Currently, there are approximately 136,000 patients in China.¹, with hemophilia A accounting for approximately 80%-85% of the total cases¹. Its symptoms mainly manifest as bleeding in the joints, muscles, and deep tissues, or hemorrhage in internal organs such as the gastrointestinal tract and central nervous system. If patients experience recurrent bleeding and do not receive timely treatment, it can lead to joint deformities or the formation of pseudotumors; in severe cases, it may be life-threatening.²Unlike many genetic disorders, patients with hemophilia can achieve “zero bleeding” and return to a normal life through long-term, regular, and standardized treatment.

However, given the actual situation in China, low-dose standardized treatment regimens are currently widely adopted. Clinical practice has shown that although this regimen can significantly reduce bleeding in patients, it does not reduce the incidence of joint lesions, and bleeding episodes still occur.²With the widespread adoption of standardized prophylactic therapy, the 2020 World Federation of Hemophilia guidelines indicate that prophylaxis should aim for higher trough level targets, ultimately striving for "zero bleeding." At a trough level of 1%, patients still face a risk of bleeding; therefore, most clinicians prefer to achieve higher trough levels (>3%–5% or higher).³In this regard, the Chinese Guidelines for Hemophilia Treatment (2020 Edition) recommend that optimal individualized treatment plans be developed based on the patient’s age, bleeding phenotype, pharmacokinetic (PK) characteristics, and lifestyle, with adjustments to the dose, concentration, and frequency of clotting factor injections to effectively prevent undertreatment or overtreatment.⁴。

Professor Yang Renchi, Director of the Center for Diagnosis and Treatment of Thrombosis and Hemostasis, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

"In recent years, with the innovation and upgrading of medical technology and treatment fields, China's hemophilia diagnosis and treatment work has made significant progress, transitioning from traditional on-demand treatment methods to a more proactive and personalized treatment model."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“He stated, ‘The essence of prophylactic therapy for hemophilia lies in long-term, regular replacement therapy. I am delighted to see the approval of China’s first pharmacokinetics-guided management tool, which will provide personalized and sustainable treatment management for every patient, helping them break free from the constraints of the disease and enjoy a life of freedom.’”

myPKFiT®BeneFIX under guidance^®(Recombinant Human Coagulation Factor VIII for Injection) treatment can reduce the risk of bleeding, optimize therapeutic efficacy and drug accessibility, thereby enhancing patients’ confidence in treatment and improving adherence. myPKFiT®Using validated Bayesian methods and based on more than 2,000 cases of Baiyinzhi^®User Blood Sample Data Modeling: Specific and Reliable Data. According to interim analysis data from a UK-PK study, standardized treatment guided by pharmacokinetics (PK) increased the proportion of patients with zero bleeding episodes to 50%, and patient adherence improved by 92.5%.⁵. In terms of treatment costs, a comparative study showed that PK-guided therapy reduced overall treatment costs by 10.67% compared with standard therapy.⁶。

Shan Guohong, President of Takeda China

Shan Guohong, President of Takeda Chinastated: “Very pleased to see myPKFiT®This internationally advanced, personalized hemophilia treatment management software has been approved in China, providing Chinese patients with customized disease management solutions to help them return to normal life. As a global leader in the field of rare diseases, Takeda boasts a long-standing heritage and extensive experience in hemophilia, consistently spearheading the advent of a new era in personalized hemophilia treatment. Looking ahead, Takeda will uphold its core philosophy of “Patients First,” closely addressing the urgent unmet needs of rare disease patients in China. The company will accelerate the introduction of more first-in-class and best-in-class innovative therapies, continuing to contribute to improving the diagnosis and treatment landscape for rare diseases in China and enhancing patients’ quality of life.

For more information, please visit the website:https://www.mypkfit.cn/

1、 The Prevalence of Hemophilia in Mainland China: a systematic review and meta-analysis, Southeast Asian J Trop Med Public Health. 2014 Mar;45(2):455-66

2. Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 Edition), People's Medical Publishing House, 2019, p. 177

3. Ushering in a New Era of Personalized Treatment for Hemophilia, Frontiers in Hematology, October 10, 2020, https://mp.weixin.qq.com/s/CyA2ixwY1xeo03qkZ10lNA

4. “Chinese Guidelines for the Treatment of Hemophilia (2020 Edition),” Chinese Journal of Hematology, 2020, 41 (2020-04-16).

5、UKPK trial interim analysis: Use of Pharmacokinetic intervention to optimise Factor VIII prophylaxis in severe haemophilia A，https://www.postersessiononline.eu/173580348_eu/congresos/WFH2018/aula/-T-MP_87_WFH2018.pdf

 6、PK-driven prophylaxis versus standard prophylaxis: When a tailored treatment may be a real and achievable cost-saving approach in children with severe hemophilia A，https://pubmed.ncbi.nlm.nih.gov/28692842/