Takeda Announces Inclusion of Two Innovative Rare Disease Therapies in China's 2021 National Reimbursement Drug List

Beijing, December 3, 2021 /PR Newswire/ -- On December 3, 2021, Takeda China announced the launch of two innovative drugs in the rare disease field, Replagal^®(Recombinant Human Agalsidase Alfa Injection Concentrate) and Fazao优^®(Injectable Icatibant Acetate) Successfully Included in the "National Basic Medical Insurance, Work-related Injury Insurance, and Maternity Insurance Drug Catalog (2021)" (hereinafter referred to as the 2021 National Medical Insurance Drug Catalog). These two innovative drugs for rare diseases were rapidly included in the medical insurance within just one year of entering China, which helps further promote the clinical practice and popularization of standardized diagnosis and treatment for rare diseases, allowing more Chinese patients with Hereditary Angioedema and Fabry Disease to benefit from innovative treatment options.

President of Takeda China, Guohong ShanSaid: "We are very grateful for the relentless efforts made by the Chinese government to improve the accessibility and affordability of innovative drugs. The inclusion of more innovative drugs in the field of rare diseases in this medical insurance catalog adjustment reflects the country's high attention and support for the vast number of patients with rare diseases and their families. It also shows the guiding principle of relevant government departments in supporting drugs with significant clinical needs and high value during the process of adjusting the medical insurance catalog. Adhering to the concept of ‘putting patients first,’ Takeda is making every effort with sincerity to actively cooperate with the government’s various medical security initiatives, contributing to enhancing the accessibility of innovative drugs for patients with rare diseases and benefiting these patients and their families."

In recent years, China has introduced a series of policies and measures to expand the inclusion of innovative drugs in the medical insurance coverage, making significant progress particularly in improving the accessibility of innovative drugs for rare diseases. The scope of adjustments to the National Medical Insurance Drug List in 2021 shows that more attention has been given to areas of major diseases such as rare diseases.

Among the newly included innovative drugs for rare diseases in the medical insurance catalog this time, Replagal^®(Agalsidase Alfa Injection Concentrate) is an enzyme replacement therapy derived from human cells and produced in a human cell line.^[1], used for the long-term treatment of Fabry disease patients. Fabry disease is a rare X-linked hereditary lysosomal storage disorder. If patients do not receive effective treatment, their symptoms will gradually worsen and lead to organ damage in the kidneys, heart, and other organs, and may even be life-threatening. Currently, enzyme replacement therapy is recommended as the first-line treatment for Fabry disease by guidelines/consensus both in China and internationally.^[2]。

Professor Chen Nan, Chief Physician of the Department of Nephrology, Ruijin Hospital, Shanghai Jiao Tong University School of MedicineSaid: "If Fabry disease patients cannot receive specific treatment in time, the progression of the disease can lead to multi-organ damage and even life-threatening complications. Therefore, initiating standardized, full-dose enzyme replacement therapy as early as possible plays a crucial role in improving the patient’s condition and preventing severe complications. It is encouraging to see that relevant treatment drugs have been quickly included in medical insurance, bringing new hope for improving patient survival rates and quality of life, allowing more Chinese Fabry disease patients to achieve long-term standardized treatment sooner and reintegrate into society."

Another innovative rare disease drug included in the medical insurance is Firazyr® (Icatibant Acetate Injection), which is the first and currently only one of its kind in China.^[3]The only selective antagonist of bradykinin 2 (B2) receptor for subcutaneous treatment of acute attacks of hereditary angioedema (HAE)^[4], which can help patients promptly manage acute HAE attacks and reduce the risk of fatal asphyxiation caused by laryngeal edema. According to statistics, 58.9% of HAE patients in China have experienced laryngeal edema, with a mortality rate as high as 40%.^[2], one of the main causes of death for HAE patients. Previously, due to the lack of targeted treatment for acute HAE attacks in China, patients could only rely on infusions of freeze-dried fresh plasma to reduce edema. This method has many limitations, and the plasma itself contains plasma proteases and substrates that cause edema, which may worsen angioedema.^[5]。

Professor Zhi Yuxiang, Department of Allergy, Peking Union Medical College Hospital, Chinese Academy of Medical SciencesSaid: "Chinese HAE patients have long lacked targeted treatment drugs for acute attacks, living in constant fear as edema attacks in various parts of the body severely affect their quality of life. In particular, acute laryngeal edema attacks can even be life-threatening. Therefore, we are very pleased that Icatibant Acetate Injection has been included in the medical insurance, providing a timely solution for the treatment of acute attacks, and taking a crucial step towards the treatment goal of 'zero attacks'."

As a global innovative biopharmaceutical company that prioritizes patients, is values-based, and driven by research and development, Takeda Pharmaceutical Company Limited remains committed to the Chinese market. By 2025, the company will launch over fifteen innovative products in China, accelerating the delivery of innovative medicines to Chinese patients with complex and rare diseases. Upholding its commitment to Chinese patients, Takeda aims to address their unmet medical needs.

Approval No.: C-ANPROM/CN/FIR/0040
Approval Date: November 2021

[1] Replagal® Prescription Information

[2] Expert Consensus on the Diagnosis and Treatment of Fabry Disease in China, Chinese Medical Journal, January 22, 2013, Vol. 93, No. 4

[3] As of November 2021

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

[5] Yao Xiaojian, Qin Kun, Zhang Tangde. Research Progress in the Prevention and Treatment of Hereditary Angioedema[J]. Journal of Dermatology and Venereology, 2018, 25(1):53-56 DOI:10.3969/j.issn.1674-8468.2018.01.014.