Takeda Showcases Robust Rare Disease Pipeline with Five Innovative Therapies at CIIE

Shanghai, November 7, 2020 /PRNewswire/ -- Today, Takeda Pharmaceutical Company Limited held a special press conference themed "Standing Together for Rare Lives" at the 3rd China International Import Expo (CIIE), unveiling five new rare disease treatments. These innovations span therapeutic areas including Fabry disease, hereditary angioedema, Gaucher disease, and hemophilia. The company also officially announced the name of its patient organization empowerment initiative in China, "Breaking the Cocoon into a Butterfly," along with its phased achievement, the "China Patient Organization Growth Map." These initiatives underscore Takeda's firm commitment to collaborating with all sectors to build a patient-centric innovative ecosystem for rare diseases in China and to drive the robust development of the country's rare disease cause.

“We are deeply grateful to the government for providing such a broad platform as the China International Import Expo (CIIE), enabling countries, industries, and enterprises to showcase cutting-edge technologies and innovative products, promoting the integration of global trade and innovation, and fully demonstrating the government’s determination to advance mutual openness and foster innovation-driven development.”Mr. Shan Guohong, Global Senior Vice President of Takeda Pharmaceutical Company Limited and President of Takeda China“As a multinational pharmaceutical company dedicated to R&D innovation, we look forward to leveraging the China International Import Expo (CIIE) to showcase Takeda’s innovative medicines and breakthrough therapies in our core therapeutic areas, accelerate the introduction of more cutting-edge international drugs, help Chinese patients access treatments synchronized with global standards, ensure that rare disease patients in China have access to effective therapies, and enable them to return to healthy lives.”

Shan Guohong, Senior Vice President of Takeda Pharmaceutical Company Limited and President of Takeda China, Delivered the Keynote Address

Rare diseases, also known as orphan diseases, refer to a wide range of conditions with very low prevalence. Currently, the World Health Organization (WHO) recognizes more than 7,000 rare diseases, 80% of which are genetic disorders, and only 5% have effective treatments or control measures. Although each individual rare disease is uncommon, the large number of different conditions results in a significant patient population; it is estimated that there are over 20 million people with rare diseases in China.^[1], most face numerous challenges such as low diagnosis and treatment rates, high misdiagnosis rates, and limited drug accessibility.

Takeda is committed to translating scientific achievements into highly innovative, breakthrough therapies that benefit patients in China and around the world. Leveraging its innovation capabilities and local expertise, Takeda is dedicated to building a robust rare disease pipeline, aligning the needs of Chinese patients with its global drug development strategy to achieve simultaneous global new drug development. In 2020 alone, Takeda expected to launch nearly seven innovative drugs, more than half of which were focused on the rare disease sector.

Taking hereditary angioedema (HAE) as an example, this is a rare genetic disorder that threatens patients' lives and can cause acute edema in multiple parts of the body without warning. Among these, laryngeal edema is the most life-threatening, as it can rapidly lead to breathing difficulties or suffocation.^[2]. According to statistics, 58.9% of HAE patients in China have experienced laryngeal edema^[3], with a case fatality rate as high as 11%–40%^[2], posing a serious threat to patients' lives. Due to the significant side effects of traditional medications, many diagnosed patients refuse treatment, leaving them in a long-term predicament with no therapeutic options.

At this year’s CIIE, Takeda unveiled for the first time two innovative therapies for hereditary angioedema (HAE) slated for introduction into China, including lanadelumab injection—the only therapy worldwide currently approved for prophylactic treatment of HAE—which reduces the frequency of recurrent edema attacks in patients and prevents asphyxiation caused by life-threatening laryngeal edema.^[4], improving the quality of life for patients and their families, and filling the gap in China where there had long been no targeted treatment for HAE. Another drug, icatibant acetate injection for acute attacks of HAE, has directly submitted a marketing authorization application in China and has entered priority review.

“The CIIE is a major event that captures the attention of the entire Chinese population. As healthcare professionals, we closely follow and eagerly anticipate the showcase of more internationally leading innovative therapies at the CIIE. For rare diseases such as hereditary angioedema, it is estimated that there are nearly 30,000 patients in China who have long lacked effective treatment and face life-threatening risks at all times.”Professor Zhi Yuxiang, Department of Allergy, Peking Union Medical College Hospital, Chinese Academy of Medical SciencesHe stated, “I am delighted to see innovative therapies for hereditary angioedema (HAE) showcased at the China International Import Expo. This signifies that the aspiration of HAE patients to achieve ‘zero’ angioedema attacks is within reach. According to existing research literature, lanadelumab provides long-term prophylaxis, with approximately 70%–80% of patients achieving complete freedom from attacks after a period of treatment. This represents a tremendous boon for HAE patients in China.”

While accelerating the introduction of innovative therapies and personalized disease solutions, Takeda remains true to its original aspiration, consistently upholding its core patient-centric philosophy. Over the years, Takeda has worked closely with rare disease patient organizations in China to achieve mutual growth and progress. This September, Takeda China announced its support for the initiative launched by the Wuxi Lingshan Charity FoundationChina Rare Disease Patient Organization Empowerment Project, aiming to help patient organizations enhance their professionalism and maturity, and further promote the vigorous development of the cause of rare diseases in China.

At this special press conference on rare diseases, Takeda, representing the Wuxi Lingshan Charity Foundation, unveiled the official name of the China Rare Disease Patient Organization Empowerment Project—“Breaking the Cocoon into a Butterfly”—along with its interim achievement, the “Growth Map for Chinese Patient Organizations.” This initiative symbolizes the project’s commitment to helping patient organizations identify their current stage of development, recognize the characteristics of each phase, precisely clarify developmental needs and key priorities at every stage, and cultivate core competencies. Ultimately, it aims to empower patient organizations to assume an increasingly vital role in the advancement of rare disease care in China.

Launch Ceremony of the Growth Map for Chinese Patient Organizations

[1] National Health Commission of the People’s Republic of China, Office of the Expert Committee on Diagnosis, Treatment, and Assurance for Rare Diseases (Peking Union Medical College Hospital). Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 Edition). 2019;5.

[2] National Health Commission, Office of the Expert Committee on Diagnosis, Treatment, and Assurance for Rare Diseases (Peking Union Medical College Hospital). Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 Edition). 2019, 190.

[3] Xu YY, Zhi YX, Liu RL, et al．Upper airway edema in 43 patients with hereditary angioedema [J]．Ann Allergy Asthma lmmunof，2014, 112(6): 539-544.

[4] Riedl M A , Maurer M , Bernstein J A , et al. Lanadelumab demonstrates rapid and sustained prevention of hereditary angioedema attacks[J]. Allergy, 2020.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (Tokyo Stock Exchange Ticker Symbol: 4502(NYSE: TAK) is a Japan-based, values-driven, R&D-focused global biopharmaceutical company. Takeda is committed to translating scientific discoveries into highly innovative medicines that safeguard patients’ health and well-being for a better future. Takeda focuses its drug development efforts on four core therapeutic areas—oncology, gastroenterology, neuroscience, and rare diseases—with dedicated investments in plasma-derived therapies and vaccines. We remain steadfastly focused on developing highly innovative medicines by pioneering novel treatment approaches and strengthening our collaborative R&D engine to build a robust and diverse product pipeline that helps improve people’s lives. With employees across approximately 80 countries and regions, we work hand-in-hand with local healthcare partners to deliver health benefits to patients worldwide.

Takeda entered China in 1994. Takeda China’s headquarters is located in Shanghai, with its main business centers on the Chinese mainland situated in Beijing, Shanghai, Tianjin, Guangzhou, and the Hong Kong Special Administrative Region, among other cities and areas. The company has established offices in major cities across China and currently employs more than 2,000 people in the country. 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 visithttps://www.takeda.com

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