Takeda China Hosts the 2nd Takeda China Pediatric Summit, Launching New Therapies for CPP, Fabry Disease, and Gaucher Disease

SHANGHAI, June 26, 2021 /PRNewswire/ -- Today, the "Guarding Childhood Dreams, United for Rare Disease Care" 2nd Takeda China Pediatric Summit, hosted by Takeda China, officially opened. The summit brought together over 500 pediatric professionals from China and abroad to discuss cutting-edge topics, including the current diagnostic and treatment landscape and the latest therapeutic options for pediatric diseases such as Central Precocious Puberty (CPP), pediatric Fabry disease, and Gaucher disease. With a focus on promoting early disease intervention and early diagnosis and treatment during critical developmental periods, the event called for collaborative efforts to build an innovative child health service system and advance the diagnosis and treatment of pediatric diseases in China.

“Children's Dream Sky, Together Safeguarding Rare Diseases” The 2nd Takeda Pediatric Summit Grandly Held

Children are the future of the nation and its people, and children's health serves as a crucial cornerstone of the "Healthy China" initiative. The 14th Five-Year Plan explicitly states that "improving the children's health service system and preventing and controlling childhood diseases" shall be prioritized as one of the key tasks.^[1]. However, pediatric health management and disease diagnosis and treatment in China still face challenges. Taking CPP as an example, this condition severely affects children's healthy growth, with its incidence rate rising steadily year by year in recent years.^[2]。In the field of rare diseases such as Fabry disease and Gaucher disease, affected children and their families commonly face challenges including low disease awareness, low diagnosis rates, and low treatment rates, which often results in missing the optimal window for timely diagnosis and intervention.

In response to the aforementioned challenges, numerous experts at this forum engaged in in-depth discussions on how to enhance the scientific and technological capabilities for promoting healthy growth and development and disease prevention and control among children in China.Professor Ding Jie of Peking University First Hospital, a member of the National Committee of the Chinese People's Political Consultative Conference (CPPCC), stated:“Since the reform and opening-up, China has achieved remarkable success in children's health management and the prevention and treatment of pediatric diseases. However, compared with international leading standards, there is still a long way to go. In disease domains that severely impact children's healthy growth—such as common, high-incidence, and rare pediatric diseases—we must further strengthen basic research and the capacity for translating scientific achievements into clinical applications, improve the pediatric talent cultivation system, and establish institutional mechanisms for healthy development tailored to the physiological characteristics of Chinese children, thereby driving the high-quality advancement of children's health management in China.”

Pediatric experts in China and abroad emphasize that for many pediatric diseases, early intervention, early diagnosis, and treatment during the critical period of child development are crucial. Taking CPP as an example, the premature development of secondary sexual characteristics and early sexual maturation may lead to associated psychological issues or abnormal social behaviors in affected children.Group Leader of the Endocrinology, Genetics and Metabolism Group, Pediatric Branch of the Chinese Medical Association, National Clinical Research Center for Child Health and Diseases, Executive Vice President of the Children's Hospital, Zhejiang University School of Medicine, Professor Fu Junfenstated: "CPP has a significant impact on both the physical and psychological health of affected children. Fortunately, in China, the treatment of CPP has now established routine and standardized diagnostic and therapeutic protocols. Current domestic and international research findings and clinical practice indicate that the use of GnRHa medications can effectively suppress pubertal development. Timely treatment can delay bone age progression in pediatric patients with CPP, ultimately improving their final adult height, while simultaneously preventing early menarche and averting associated adverse psychological and behavioral problems."

With growing attention from all sectors of society toward rare diseases in recent years, the diagnosis and management of pediatric rare diseases has emerged as a key topic at this conference. Attending experts engaged in a series of discussions on diagnostic and therapeutic strategies for childhood-onset rare diseases, represented by Fabry disease and Gaucher disease. Fabry disease is a rare X-linked inherited lysosomal storage disorder. Patients experience severe burning pain in their hands and feet, accompanied by hypohidrosis, anhidrosis, and heat intolerance. As the disease progresses, symptoms gradually worsen and lead to organ pathology in the kidneys, heart, and other systems, such as severe renal failure progressing to uremia, or cardiovascular and cerebrovascular complications like stroke and myocardial infarction. Without effective treatment, the condition will become life-threatening.^[3]。Professor Xu Hong, Chief Physician and Doctoral Supervisor, Children's Hospital of Fudan University.stated: “Patients with Fabry disease often present with clinical symptoms during childhood and adolescence, and these symptoms progressively worsen over time. Failure to initiate early treatment significantly increases the risk of progressive multisystem disease. Initiating enzyme replacement therapy in childhood can effectively alleviate the clinical symptoms of Fabry disease and delay or prevent progressive organ damage. Therefore, for these pediatric patients, early diagnosis and treatment must be highly prioritized.”

Another rare disease that garnered significant attention at the conference was Gaucher disease. Caused by a deficiency of glucocerebrosidase, the condition prevents the degradation of glycolipids, leading to their systemic accumulation in multiple organs. Patients typically present in childhood with moderate-to-severe hepatosplenomegaly, anemia, moderate-to-severe thrombocytopenia, and skeletal involvement. Severe cases may progress to splenic infarction, splenic rupture, or even life-threatening hemorrhage. Additionally, approximately 30%–80% of patients with childhood or adolescent onset experience growth and developmental impairment; the earlier the onset, the more pronounced the linear growth deficit.^[4]。Professor Luo Xiaoping, Vice Chairman of the Pediatric Branch of the Chinese Medical Association and Director of the Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technologystated: "The clinical manifestations of Gaucher disease progressively worsen, are disabling, and life-threatening. Fortunately, Gaucher disease is one of the few rare diseases for which disease-specific pharmacological treatments are available. Taking Gaucher disease as an example, enzyme replacement therapy (ERT) can continuously improve clinical signs such as hepatosplenomegaly, anemia, thrombocytopenia, and skeletal involvement in patients. For pediatric patients, timely initiation and long-term adherence to enzyme replacement therapy can improve and maintain growth and development, enabling them to return to a normal life."。”

As the initiator of the forum, Takeda China is committed to leveraging its extensive expertise and accumulated resources, and partnering with all stakeholders to deliver high-quality disease management solutions for more pediatric patients, safeguarding the healthy lives of children in China.Mr. Guohong Shan, President of Takeda Chinastated: "As a values-based, R&D-driven, and patient-centric global innovative pharmaceutical company, pediatric health remains one of Takeda’s sustained key focus areas globally. Since entering China 27 years ago, we have been working closely with stakeholders across all sectors of society to actively explore the pediatric field and continuously accelerate the introduction of innovative medicines. We are also strengthening academic exchange and collaboration among all parties, pooling efforts to explore new approaches to pediatric disease management, and safeguarding the healthy growth of children in China."

Shan Guohong, President of Takeda China

At this forum, Takeda announced the official introduction to China of Enantone® (leuprorelin acetate microspheres for injection), a 3-month sustained-release long-acting formulation for CPP treatment. This will provide a more convenient option for the standardized and continuous treatment of pediatric CPP patients in China, helping to further improve treatment adherence and satisfaction among children, and enhance the quality of life for their families. In the rare disease field, Takeda’s enzyme replacement therapy drug for Fabry disease, Replagal®^®(Agalsidase alfa concentrated solution for injection) was officially launched in China in April this year. Additionally, VPRIV® (velaglucerase alfa for injection), an innovative therapeutic drug for Type 1 Gaucher disease, has also been approved for entry into China and is expected to be commercially available in the near future. Furthermore, Takeda China has collaborated closely with all sectors of society to actively participate in and successfully support multiple children's public welfare projects across China, contributing to the physical and mental well-being of children in China. These initiatives include supporting the China Children and Teenagers' Fund in organizing three consecutive children's summer camps to encourage pediatric patients with rare diseases to foster a spirit of scientific inquiry and a positive, optimistic attitude toward life; supporting the China Gaucher Disease Association and the Pain and Illness Challenge Foundation in launching a child psychology co-creation project to develop a parent-child interactive toolkit for Gaucher disease; and supporting the China Primary Health Care Foundation in initiating the "Patient Education Program for Children with Central Precocious Puberty in China," which utilized charity classes to help children with CPP receive standardized treatment during the pandemic.

^[1]《Outline of the 14th Five-Year Plan for National Economic and Social Development and the Long-Range Objectives Through the Year 2035》,http://www.gov.cn/xinwen/2021-03/13/content_5592681.htm

^[2]Writing Group for the Survey on Pubertal Development, Subspecialty Group of Endocrinology, Hereditary and Metabolic Diseases, Pediatric Branch, Chinese Medical Association, Chinese Journal of Endocrinology and Metabolism. 2010.26

^[3]Chinese Fabry Disease Expert Collaborative Group, National Medical Journal of China, 2013, 93(4): 243-247

^[4]Expert Consensus on Multidisciplinary Diagnosis and Treatment of Gaucher Disease (2020), National Medical Journal of China, June 23, 2020, Vol. 100, No. 24

Statement:

1. This article is intended to convey cutting-edge medical and pharmaceutical information and does not constitute a recommendation or promotion of any drug or treatment regimen.
2. If you would like to learn more about diseases or obtain information regarding medications, diagnosis, and treatment, please consult a healthcare professional.