Takeda Launches myPKFiT®, China's First NMPA-Approved Pharmacokinetic-Guided Management Tool for Hemophilia A

Kunming, December 19, 2020 /PRNewswire/ -- Takeda China today announced myPKFiT, its pharmacokinetics (PK)-guided management tool for hemophilia A (congenital factor VIII deficiency)^®Officially Launched,For Familiarity with HemophiliaFor use by healthcare professionals (HCPs) practicing Treatment A,Assist patients aged 16 years and older (weighing 45 kg or more) with hemophilia A in receiving Benefix^®Prophylactic treatment with (Recombinant Human Coagulation Factor VIII for Injection). myPKFiT^®is the first and currently only NMPA-approved management tool supporting pharmacokinetics (PK)-guided therapy for hemophilia A in China, with Advate^®When used in combination, it can reduce the risk of bleeding, optimize patients’ treatment outcomes and medication accessibility, enhance treatment confidence and improve adherence, thereby helping patients achieve the therapeutic goal of “zero bleeds.” Currently, myPKFiT^®Launched in over 50 countries and regions worldwide, registered for use by nearly 1,100 medical institutions, and benefiting more than 8,000 patients globally.

myPKFiT Launch Ceremony

Significant Unmet Needs in the Diagnosis and Treatment of Hemophilia in China: Urgent Need for Innovative Therapies to Help Patients Achieve “Zero Bleeding””

There are 136,000 hemophilia patients in China, of whom 80% have hemophilia A.-85%^[1]Due to limited awareness and financial burden, most patients currently still commonly adopt low-dose treatment regimens. Clinical practice has shown that this regimen does not reduce joint lesions and bleeding episodes still occur. If patients experience recurrent bleeding and do not receive timely treatment, it may lead to joint deformity or pseudotumor formation, which can be life-threatening in severe cases.^[2]In fact, patients with hemophilia only need to receive long-term, regular, and standardized treatment to effectively reduce the frequency of bleeding episodes, achieve “zero bleeding,” and return to a normal life.

However, due to variations in bleeding tendencies, lifestyles, and drug metabolism rates among patients, a uniform treatment regimen based solely on dose adjustments fails to meet the needs of every individual. In recent years, personalized treatment strategies have gained increasing attention, as reflected in the latest guidelines from the World Federation of Hemophilia^[3]Both emphasize the importance of individualized prophylactic treatment, as highlighted in the "Chinese Guidelines for Hemophilia Treatment (2020 Edition)."

Individualized treatment regimens are based on pharmacokinetics, optimizing factor VIII dosing and administration intervals to maintain sufficient hemostatic FVIII levels. This approach comprehensively considers multiple factors, including the patient’s bleeding phenotype (e.g., age at first joint bleed), pharmacokinetic characteristics of the clotting factor, daily activity levels, anticipated bleeding risks, presence of arthropathy, and patient adherence, thereby effectively preventing under- or over-treatment.^[4]and improve patients' quality of life^[5]。

myPKFiT^®In combination with BaiyinZhi^®(Recombinant Human Coagulation Factor VIII for Injection) Use, Helping Patients Achieve "Zero Bleeding"

myPKFiT^® Modeling Based on Large-Sample Data Including Chinese Patients and Using Validated Bayesian Methods^{[6], [7]}By collecting only two blood samples, pharmacokinetic parameters for each patient can be accurately and intelligently calculated, helping healthcare professionals develop individualized treatment plans and monitor patients’ factor levels, thereby enabling patients to better plan their daily activities. According to interim analysis data from the UK-PK study, standardized treatment guided by pharmacokinetics increased the proportion of patients with zero bleeds to 50% and improved patient adherence by 90%.^[8]. In terms of treatment costs, a comparative study showed that PK-guided therapy can reduce the number of infusions by 30% compared to standard treatment.^[9], reducing overall treatment costs by 10.67%^[10]。

“Traditional PK analysis faces significant challenges in clinical application, not only due to cumbersome sampling procedures but also because the time required for FVIII levels in patients to decline to a specific trough level varies, making assessment difficult.”Professor Yang Renchi, Director of the Thrombosis and Hemostasis Diagnosis and Treatment Center, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical SciencesmyPKFiT^®It can objectively guide the formulation of individualized treatment plans based on PK analysis results, preventing recurrent joint bleeding or disability caused by under-treatment, helping patients achieve the treatment goal of “zero bleeds” and live like normal individuals.”

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

“myPKFiT^®It is the first medical software for individualized management of hemophilia A approved by the National Medical Products Administration, marking a milestone in the development of diagnosis and treatment in the field of hemophilia in China. Hemophilia is a lifelong condition that requires whole-life-cycle disease management tailored to each patient’s specific circumstances.”Mr. Shan Guohong, President of Takeda ChinaIndications:“myPKFiT^®Its market launch not only propels China’s hemophilia field into a new era of precision and digitalized therapy, but also empowers Chinese patients to move with ease and enjoy a life of freedom. Looking ahead, Takeda will continue to leverage its advanced technological advantages and extensive experience accumulated in the hemophilia sector, actively collaborating with all stakeholders to drive the dual upgrade of diagnostic and therapeutic standards as well as medical services for hemophilia in China.”

Mr. Shan Guohong, President of Takeda China, Delivers Opening Remarks

In the future, myPKFiT^®The patient management portal, expected to be launched soon, will enable patients to better understand their internal factor levels, thereby further enhancing treatment confidence and improving adherence.

Vigorously Promote Prophylactic Treatment to Further Improve the Quality of Life for Hemophilia Patients in China

[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 Therapy 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] Vigorously promote prophylactic treatment to further improve the quality of life for patients with hemophilia, 2015 Jul;36(7),https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7342648/

[6] myPKFiT™ Device User Manual. Version 2.0.

[7] Björkman S et al. 2012.

[8] 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

[9] A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management, https://onlinelibrary.wiley.com/doi/10.1111/j.1538-7836.2011.04611.x

[10] 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/