Takeda haemophilia management tool myPKFiT was approved

Takeda China announced today that myPKFiT® a management tool for type A haemophilia (lack of congenital coagulation factor VIII) under the guidance of Pharmacodynamics (PK) is approved by the State Drug Administration (NMPA) for patients aged 16 years and older (weight 45 kg and above) who receive treatment with 100in stop® (injected recombinant human coagulation factor VIII). myPKFiT® is China's first management tool to support haemophilia drug generation dynamics (PK) guidance, but also to date the State Drug Administration's only approved individual prevention FVIII dose guidance software for medical professionals familiar with haemophilia A treatment (HCP) use. Bain stop ® and myPKFiT® can be used in combination with each patient's different characteristics and needs, individual adjustment of hemophilia patients' blood clotting octogenesia (FVIII) dosing, for China's haemophilia patients to open a new era of individualized treatment, to help achieve a free life.Hemophilia is a recessive hereditary rare disease, mainly due to the lack of coagulation factors in the patient's body leading to clotting disorders. At present, there are about 136,000 patients in China1, of which type A haemophilia accounts for about 80%-85% of the total1. The symptoms are mainly bleeding from joints, muscles and deep tissues, or internal organ bleeding such as the gastrointestinal tract and central nervous system. If the patient repeatedly bleeding and not treated in time, it will lead to joint malformation or false tumor formation, serious can be life-threatening2. Unlike many genetic diseases, haemophilia patients only need to receive long-term regular standardized treatment, it is expected to achieve "zero bleeding" and return to normal life.However, based on the actual situation in China, low-dose standard treatment is widely used. Clinical practice shows that although the program can significantly reduce bleeding in patients, but can not reduce the occurrence of joint lesions and there is still bleeding 2. The 2020 World Hemophilia guidelines say preventive treatment should set a higher valley level target and ultimately pursue "zero bleeding". Patients still have a risk of bleeding at a valley level of 1%, so most clinicians tend to reach higher valley levels (>3%-5%, or higher)3. In this regard, the China Guidelines for hemophilia Treatment (2020 edition) recommend that the optimal individualization scheme suitable for patients can be formulated according to the patient's age, bleed esopaedic gaze, pharmacogenesital dynamics (PK) characteristics and lifestyle, and adjust the dose, concentration and frequency of injection coagulation factors to effectively avoid undertreated or overtreated conditions4.“ In recent years, with the innovation of medical technology and treatment, China has made great progress in the diagnosis and treatment of haemophilia, and is changing from traditional on-demand treatment to a more active and individual treatment model. "The essence of hemophilia prevention and treatment is the need for a long-term, regular alternative treatment, " said Professor Yang Renchi, director of the thrombosis treatment center at the Hematology Hospital of the Chinese Academy of Medical Sciences. I am very pleased to see the approval of China's first management tool under the guidance of pharmacodynamics, which will provide individual, sustainable treatment management for every patient, helping patients to get out of the shackles of disease and enjoy a free life. myPKFiT® under the guidance of the bain stop ® (injection with recombinant human coagulation factor VIII) treatment, can reduce the risk of bleeding, optimize the effectiveness of treatment and drug access, thereby improving patients' confidence in treatment, improve compliance. MyPKFiT ® using the verified Bayes method and modeling more than 2,000 100-in-the-box ® user blood sample data, the data is single-to-one and reliable. According to the interim analysis data of a UK-PK study, the standardized treatment under PK's guidance increased the proportion of patients with zero bleeding to 50% and the compliance of patients by 92.5%.5%. In terms of treatment costs, according to a comparative study, treatment under PK guidance can reduce the overall cost of treatment by 10.67 percent compared to standard treatment6."It's great to see myPKFiT® an internationally advanced personalized treatment management software for haemophilia approved in China to provide Chinese patients with customized disease management programs to help them return to normal life," said Shan Guohong, President of Takeda China. As a global leader in the field of rare diseases, Takeda has a long history and accumulated experience in the field of haemophilia, and has always actively led the arrival of a new era of individualized treatment of haemophilia. In the future, Takeda will uphold the core concept of 'patient first', pay close attention to the urgent needs of patients with rare diseases in China, accelerate the introduction of more of the world's first and best-in-class innovative therapies, and continue to contribute to improving the status quo of rare disease diagnosis and treatment in China and improving the quality of life of patients. "
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Sina Medical News
)reference1. 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. Guide to Rare Diseases (2019), People's Health Press, 2019, 177 3. Opening a New Era of Individualized Treatment of Hemophilia, Blood Frontier, 10 October 2020, 4. China Guide to Hemophilia Treatment (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,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,