Home Takeda China and Chinese Red Cross Foundation Announce Strategic Collaboration to Advance Rare Disease Ecosystem in China

Takeda China and Chinese Red Cross Foundation Announce Strategic Collaboration to Advance Rare Disease Ecosystem in China

Jun 28, 2021 16:41 CST Updated 16:41
Takeda

Biopharmaceutical Manufacturer

BEIJING, June 28, 2021 /PRNewswire/ -- Takeda China and the Chinese Red Cross Foundation announced a strategic partnership in Beijing. Through a series of comprehensive solutions, both parties will jointly promote industry development in rare diseases such as Fabry disease, Gaucher disease, and hereditary angioedema, help improve the accessibility of innovative medicines, optimize full-course disease management for patients, and advance the development of care and diagnosis-and-treatment systems for rare disease patients in China.

On the same day, Takeda China announced the official launch of its innovative drug TAKHZYRO® (lanadelumab injection) for the standard treatment of hereditary angioedema (HAE), bringing an innovative treatment option to HAE patients in China.

Mr. Bei Xiaochao, Vice Chairman and Secretary-General of the Red Cross Foundation of China; Professor Li Dingguo, Chairman of the Shanghai Rare Disease Prevention and Treatment Foundation; Ms. Wang Lin, Secretary-General of the Joint Conference of Chairs of National Rare Disease Academic Societies; Professor Yin Jia, Director of the Department of Allergy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences; Professor Zhi Yuxiang; Ms. Wang Yi'ou, Secretary-General of the Beijing Pain Challenge Foundation; Mr. Shan Guohong, President of Takeda China; and numerous other distinguished guests attended the event and delivered speeches.

Under this strategic partnership, the two parties will collaborate to develop comprehensive rare disease solutions tailored to the actual needs of patients. The cooperation will encompass six key areas: enhancing diagnostic and treatment capabilities for rare diseases; advancing the overall standard of care for rare disease patients in China; reducing the time to diagnosis for rare diseases; strengthening public and patient awareness of rare diseases; establishing a digital whole-course disease management system for rare disease patients; and jointly discussing the establishment of a dedicated fund for rare disease-related initiatives. Through a series of activities and programs, including physician education, patient education, patient assistance, and patient care initiatives, the partnership aims to foster progress in China’s rare disease sector, particularly in conditions such as hereditary angioedema, Fabry disease, and Gaucher disease. It will also improve patients’ access to innovative therapies and enhance whole-course disease management capabilities. Over the next two years, both parties will systematically advance the implementation of these initiatives, jointly driving the construction and development of the rare disease field in China.

“We are delighted to establish this strategic partnership with Takeda China to jointly carry out long-term cooperation across multiple areas, including physician training, patient education, and patient assistance.” Mr. Bei Xiaochao, Vice Chairman and Secretary-General of the Chinese Red Cross Foundation, stated: “We believe that by leveraging Takeda’s extensive experience in rare diseases and integrating it with the Chinese Red Cross Foundation’s resource platform in related fields, this collaboration is expected to further enhance the diagnosis and treatment standards for rare diseases in China, drive the development of China’s rare disease sector, deliver health benefits to patients, and contribute to the Healthy China initiative.”

As one of the enterprises with years of deep engagement in the rare disease sector, Takeda has consistently spared no effort in advancing the development and progress of this field. Since entering China 27 years ago, Takeda has continuously collaborated with all sectors of society to jointly promote public education on rare diseases such as hemophilia, lysosomal storage disorders (LSD), and hereditary angioedema (HAE). The company has also supported the establishment and development of HAE patient organizations in China, helping to raise public awareness of these conditions. Meanwhile, Takeda partners with various stakeholders to advance the standardization of diagnosis and treatment for rare diseases in China, and implements patient care and assistance programs to provide sustained support to rare disease patients across the country.

“It is a great honor to establish a long-term strategic partnership with the Chinese Red Cross Foundation to jointly advance the further upgrading of China’s rare disease diagnosis and treatment ecosystem,” stated Mr. Shan Guohong, President of Takeda China. “Guided by our ‘Patients First’ principle, Takeda remains closely focused on the unmet needs of rare disease patients in China. While dedicated to accelerating the introduction of innovative rare disease therapies, we are actively exploring comprehensive solutions for diagnosis, treatment, and patient support. We are committed to delivering holistic patient care and creating a healthy life and a brighter future for rare disease patients in China.”

China has a substantial rare disease patient population, with the current estimated total exceeding 20 million and over 200,000 new cases annually. In recent years, the Chinese government has placed high priority on the rare disease community and introduced multiple supportive policies, driving rapid development in this sector. However, due to limited public awareness and the uneven distribution of medical information and resources, rare disease patients in China continue to face numerous challenges. These include low diagnostic rates, high misdiagnosis rates, and the unavailability of approved appropriate therapeutics for certain rare diseases in China, leaving patients with inadequate access to medical care and medications. Consequently, there is a critical and urgent demand among patients for the introduction of disease-specific innovative therapies.

Rare diseases represent one of Takeda’s key therapeutic areas globally. In recent years, Takeda has continuously enhanced its rare disease R&D capabilities and product pipeline, actively aligned with China’s favorable policies supporting rare diseases, and accelerated the introduction of innovative treatment options, having already launched relevant drugs in China for the treatment of hemophilia and Fabry disease. Today, Takeda China announced the official launch in China of another rare disease therapeutic from its portfolio: Takhzyro® (lanadelumab injection), the world’s first monoclonal antibody for the standard treatment of Hereditary Angioedema (HAE). Takhzyro® (lanadelumab injection) is recommended as a first-line standard therapy for HAE by the US and Canadian HAE clinical practice guidelines [1],[2]. It works by selectively inhibiting activated plasma kallikrein in patients, thereby preventing disease attacks.

Professor Zhi Yuxiang from the Department of Allergy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences stated: “Standardized treatment is crucial for controlling attacks of hereditary angioedema. I am pleased that lanadelumab, this targeted therapeutic agent, has been introduced to China, bringing HAE patients one step closer to their goal of achieving ‘zero attacks’ of edema, and will also mark a brand new starting point for the advancement of hereditary angioedema diagnosis and treatment.”

HAE is a rare autosomal dominant genetic disorder. Due to abnormal concentrations and/or function of C1 esterase inhibitor (C1-INH) in the body, patients experience unpredictable acute edema in multiple sites, including the extremities, face, genital skin, respiratory tract, and gastrointestinal mucosa. Gastrointestinal mucosal attacks are accompanied by severe colicky pain, nausea, and vomiting, severely impacting patients' daily work, study, life, and emotional well-being. The onset of attacks in most HAE patients is unpredictable, and laryngeal attacks progress rapidly, potentially leading to fatal asphyxiation. According to statistics, 58.9% of HAE patients in China have experienced laryngeal edema [3], with a mortality rate reaching up to 40% [4], making it one of the leading causes of death among HAE patients. In May 2018, the *First List of Rare Diseases*, issued by the National Health Commission and four other departments, included HAE as one of the first 121 rare diseases [5].

Takhzyro® (lanadelumab injection) was first granted marketing approval in the United States in 2018, and received approval to enter the Chinese market in December 2020, filling the long-standing gap in targeted therapies for HAE in China.

Statement:

1. This article aims to provide cutting-edge medical and pharmaceutical information and does not constitute a recommendation or promotion of any medication or treatment regimen. 2. For further information regarding diseases, medications, or diagnostic and treatment options, please consult a healthcare professional.

# About Takeda Pharmaceutical

Takeda Pharmaceutical Company Limited (TSE: 4502) (NYSE: TAK) is a Japan-based, values-driven, and R&D-focused global biopharmaceutical company. Takeda is committed to translating scientific discoveries into highly innovative medicines to safeguard healthy lives and a brighter future for patients. The company focuses on drug development across four therapeutic areas: Oncology, Gastroenterology, Neuroscience, and Rare Diseases, with dedicated investments in Plasma-Derived Therapies and Vaccines. We remain steadfast in our focus on highly innovative medicines, pioneering novel treatment approaches and strengthening our collaborative R&D engine to build a robust and diverse pipeline that helps improve people's lives. Our employees operate across approximately 80 countries and regions, partnering with local healthcare stakeholders to deliver better health outcomes for patients worldwide.

Takeda entered China in 1994, with its China headquarters located in Shanghai. Its main business centers in mainland China are located in Beijing, Shanghai, Tianjin, Guangzhou, and the Hong Kong Special Administrative Region, along with offices in major cities across China. Currently, the company employs over 2,000 staff in China. With China's economic development and the growing demand for healthcare, China has become one of Takeda's most important emerging markets globally.

For more information, please visit https://www.takeda.com

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[1] Busse P J , Christiansen S C , Ms M A R , et al. US HAEA Medical Advisory Board 2020 Guidelines for the Management of Hereditary Angioedema[J]. The Journal of Allergy and Clinical Immunology: In Practice, 2020.

[2] Betschel S , Badiou J , Binkley K , et al. The International/Canadian Hereditary Angioedema Guideline[J]. Allergy Asthma and Clinical Immunology, 2019, 15:72.

[3] Busse P J, et al. The Journal of Allergy and Clinical Immunology: In Practice, 2021, 9(1):132-150

[4] National Health Commission, Office of the Expert Committee on Rare Disease Diagnosis, Treatment and Care (Peking Union Medical College Hospital). Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 Edition). 2019.

[5] National Health Commission, Ministry of Science and Technology, Ministry of Industry and Information Technology, National Medical Products Administration, National Administration of Traditional Chinese Medicine. Catalogue of the First Batch of Rare Diseases, Document No. 10 [2018] of the National Health Commission. http://www.nhc.gov.cn/yzygj/s7659/201806/393a9a37f39c4b458d6e830f40a4bb99.shtml