A number of Takeda's innovative drugs debuted at CIBO to promote the upgrading of the innovation ecosystem of rare diseases from products to services

SHANGHAI, Nov.
6, 2022 /PRNewswire/ -- During the 5th China International Import Expo, Takeda Pharmaceutical unveiled five innovative products at Takeda's special launch event in the field of rare genetic and blood diseases
.
Maribavir^[2], which is used for the treatment of anti-cytomegalovirus (CMV) infection or disease after transplantation^[1], ushered in the "Asian premiere", and the full range of products of the "rare coagulation innovative drug family" was unveiled, further highlighting the company's innovative advantages in the field of rare diseases and its determination and commitment
to continue to provide innovative treatment options for Chinese patients 。 In addition to accelerating the introduction of innovative products, Takeda China is committed to working with all parties in the industry to continuously upgrade the patient-centered innovation ecosystem for rare diseases, strengthen diagnosis and treatment cooperation in hereditary angioedema, hemophilia, Fabry disease and other diseases, and accurately understand patient needs through patient insights, providing patients with a full range of service support and a multi-level protection system
.

Mr.
Guohong, Shan Guohong, Senior Vice President of Takeda Pharmaceutical and President of Takeda China, said, "It is an honor to use the platform of the 5th CIIE to share Takeda's innovation achievements
in rare genetic and blood diseases.
Thanks to the 'spillover effect' of the Expo and a series of powerful measures taken by relevant national departments to encourage innovation and optimize approval and review, the speed of approval of Takeda Pharmaceutical's innovative drugs and breakthrough therapies in China continues to set new records
.
At the same time, we actively cooperate with all sectors of society to continue to promote rare disease awareness, policy advocacy, multi-party cooperation, patient care and other fields, helping patients to achieve 'medical treatment, medical medicine, and drug insurance', and escort patients with rare diseases in China
.
" "

A number of innovative drugs in the field of rare diseases were unveiled, bringing new hope for patients

Hemophilia is one of Takeda's key areas of rare genetics and blood, with more than 70 years of experience and heritage in hemophilia treatment, with a broad, diversified and highly differentiated product portfolio to provide transformative and personalized treatments
for patients with hemophilia and other blood disorders.
This year, it coincides with the 10th anniversary of the launch of Baiinzhi in China, benefiting nearly 5,000 hemophilia patients
in China.
At the same time, taking advantage of the Expo, Takeda once again made a collective appearance at the Takeda booth with the new "Rare Blood Coagulation Innovative Drug Family"
.
The "family" includes Susoctocog alfa for injection for acquired hemophilia, Vonicog alfa for injection for von Willebrand disease, Pei recombinant human coagulation factor VIII for injection for hemophilia A, and recombinant ADAMTS-13 for thrombocytopenic purpura, each of which provide new treatment options
for patients with rare coagulation diseases of different types and treatment needs.

Meanwhile, myPKFiT 3.
0[^3], another member of the "family", is the first upgraded version of a management tool in China to support hemophilia pharmacokinetics (PK)-guided ^[4].

According to an interim analysis of data from a UK-PK study, PK-guided standardized treatment increased the proportion of patients with zero bleeding to 50% and patient compliance by 90% ^[5].

The myPKFiT version 3.
0 update iteration realized the visual
monitoring of patient coagulation factor levels and interactive patient management.
Optimize the patient's treatment effect and individualized drug delivery mode to help patients achieve the treatment goal
of "zero bleeding".

At this year's CIIE, malibavir^[2], an innovative drug for post-transplant anti-CMV therapy from Takeda, ushered in an "Asian premiere"
.
Maribavir [^2] is the world's first and currently the only approved drug for the treatment of refractory (with or without drug resistance) (R/R) cytomegalovirus (CMV) infection or disease following organ transplantation or hematopoietic cell transplantation ^[6].

Studies have shown that the proportion of patients in the maribavir^[2] treatment group who achieve a defined endpoint for viral clearance is more than
twice as high as that of conventional antiviral therapy.
In addition, maribavir ² had lower
treatment-related side effects compared with conventional antiviral therapy.
^[7]

Professor Wu Depei, director of the Department of Hematology, The First Affiliated Hospital of Soochow University, said: "The treatment of postoperative infection after transplantation surgery is a common difficulty faced by hematologists in China, especially CMV infection, patients have complex conditions and poor prognosis, which need to be paid enough attention
.
Internationally, maribavir ^[2] is one of the highly anticipated innovative drugs for CMV infection, and it is expected to meet
Chinese patients as soon as possible.
"

At present, Maribavir ^[2] has been identified as an orphan drug in many countries and regions such as the United States, Europe, Australia and South Korea, and has been approved by the FDA through
priority review.
In China, it has been officially recognized as a "breakthrough therapy drug variety" by the Center for Drug Evaluation of the State Medical Products Administration, and it is expected that maribavir ^[2] will bring new therapeutic hope
to Chinese patients with CMV infection/disease.

In recent years, thanks to the spillover effect of the Expo, a number of Takeda's innovative drugs have accelerated their entry into China
.
Since the first CIIE in 2018, Takeda Pharmaceutical has approved five innovative products in the field of rare genetic and blood diseases in China, covering hemophilia, Fabry disease, Gaucher disease, hereditary angioedema and other disease areas
.
Among them, the injection of altebant acetate for the treatment of hereditary angioedema and the concentrated solution of agalase α injection for the treatment of Fabry disease were unveiled in Jinbo last year, and were included in the 2021 national medical insurance drug catalogue
in the same year.

Help improve the level of diagnosis and treatment, and work with all parties to promote the upgrading of the innovation ecosystem of rare diseases

In the field of rare genetic and blood diseases, Takeda is committed to a longer-term industry layout, actively working with all parties in the industry to empower key driving roles in the industry, and gradually forming a patient-centered rare disease innovation ecosystem that can operate autonomously, but also interconnects and promotes each other
.

Hereditary angioedema is a rare autosomal dominant disorder with a global prevalence of approximately 1 in 50,000 ^[8].

Most patients have unpredictable seizures, high frequency and moderate intensity
.
It should be noted that edema in the larynx progresses rapidly, and if the rescue is not timely, it can lead to suffocation and death
in 4.
6 hours.
According to statistics, 58.
9% of patients with hereditary angioedema in China have developed laryngeal edema [^9], and its mortality rate can reach up to 40%, which is one of the main causes of death of the disease ^[10].

In China, doctors and patients know little about hereditary angioedema, resulting in low
diagnosis and treatment rates.
In order to change this situation, this year, with the support of the China Alliance for Rare Diseases and the Beijing Society for the Diagnosis, Treatment and Security of Rare Diseases, the National Expert Committee on Hereditary Angioedema was officially established, and the first academic journal in the field of hereditary angioedema in China "New Progress in Hereditary Angioedema" was established, which further accelerated the high-quality development
of discipline research and clinical practice in the field.

At this year's CIIE, Takeda China and the China Alliance for Rare Diseases launched the "Hereditary Angioedema Standardized Diagnosis and Treatment Project", aiming to establish 100 hereditary angioedema diagnosis and treatment centers and form a diagnosis and treatment collaboration network by 2025, so as to improve clinicians' awareness of rare diseases, improve the rate of disease screening and diagnosis, shorten the path and practice of rare disease patients, help meet the actual diagnosis and treatment needs of rare disease patients and their families in China, and thus improve the quality of life
.

Mr.
Li Linkang, Executive Chairman of China Alliance for Rare Diseases, said: "How to promote the upgrading of the prevention and treatment of rare diseases so that patients can receive timely and effective diagnosis and treatment is still a challenge
for China on the road to promoting the construction of a healthy China.
We are pleased to see that China's ability to diagnose and treat hereditary angioedema diseases is far better than in the past
.
It is expected that the Alliance for Rare Diseases will work with all sectors of society to jointly promote the 'Hereditary Angioedema Standardized Diagnosis and Treatment Project', so that every patient with hereditary angioedema can receive timely and accurate diagnosis and treatment
.
" "

In recent years, under the background of the continuous advancement of the digital economy, artificial intelligence-assisted diagnosis and treatment has developed rapidly
.
The clinical decision support system (CDSS), led by Professor Zhi Yuxiang, chief physician of the Department of Allergy of Peking Union Medical College Hospital, will open up an important exploration
of digital solutions in the clinical diagnosis of hereditary angioedema.
"The CDSS system will quickly move from 'concept' to 'value', and patients with high-risk hereditary angioedema can be screened through data screening models in the clinic, reducing 'missed diagnosis', and thus empowering clinicians to make diagnosis and treatment decisions
.
" Professor Zhi Yuxiang said
.

In addition, in the field of Fabry disease, Takeda China has worked with multiple resources to build a diagnosis and treatment capacity training platform and diagnosis and treatment collaboration network, helping to improve clinicians' understanding of rare diseases, improve the rate of disease screening and diagnosis, and shorten the path and practice
of rare disease patients.
So far, the project has extended to 28 provinces across the country, covering hundreds of hospitals and hundreds of doctors involved in the clinical diagnosis and treatment of Fabry disease, and helping hundreds of patients to get early diagnosis and treatment
through the dried blood spot (DBS).

At last year's CIIE, Takeda signed the "100% Pilot" hemophilia multidisciplinary diagnosis and treatment capacity improvement project with China Rare Disease Alliance, Beijing Society for Rare Disease Diagnosis and Treatment, and China Hemophilia Collaboration Group, aiming to promote the construction of recommendations of China's hemophilia tertiary center, improve the clinical diagnosis and treatment capacity of hemophilia in China, and help patients truly achieve localized management and homogeneous treatment
.
One year after the launch of the project, more than 700 doctors have received training and counseling, which has comprehensively promoted the construction of tertiary diagnosis and treatment of hemophilia in China and the improvement
of diagnosis and treatment level.

"Putting patients first" is Takeda's long-term commitment to meeting the unmet needs of patients with rare diseases
.
At this year's CIIE, Takeda, together with representatives of China Rare Disease Alliance and patient organizations, jointly released the "Industry Research Report on Patient Registration and Patient Research by Chinese Rare Disease Patient Organizations", focusing on the exploration and achievements
of Chinese rare disease patient organizations in data registration and related research work.
At the same time, this year is also the 10th anniversary of Takeda's introduction of the first hemophilia product, and at this press conference, Takeda and the guests witnessed the company's commitment and actions
to continue to contribute to the development of China's hemophilia field for ten years, lead the concept of personalized precision treatment, and benefit more Chinese patients.

Approval number: C-ANPROM/CN/GEN/0132 Approval date: November 2022