Takeda Announces China Launch of VPRIV® (Velaglucerase Alfa) for Long-Term Enzyme Replacement Therapy in Type 1 Gaucher Disease

Shanghai, February 26, 2022 /PRNewswire/ -- Today, Takeda China announced the launch of its long-term enzyme replacement therapy (ERT) drug, Vpriv, for patients with Type 1 Gaucher disease.^®(Injectable Velaglucerasea) has been officially launched in China, bringing a new treatment option to Chinese patients with Gaucher's disease. Professor Luo Xiaoping from Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, and Professor Zhang Liansheng from the Second Hospital of Lanzhou University served as the conference chairs. Professor Wang Tianyou from Beijing Children's Hospital, Capital Medical University, and Professor Shao Zonghong from Tianjin Medical University General Hospital attended as special guests. Mr. Shan Guohong, President of Takeda China, and other guests participated both online and offline, engaging in in-depth discussions on the current state of Gaucher's disease diagnosis and treatment in China and the latest treatment experiences.

Vepure® China Launch Event - Shanghai

Gaucher's disease is a rare autosomal recessive genetic metabolic disorder, mainly caused by gene mutations that lead to a lack or reduction in the activity of glucocerebrosidase in the body. This results in the accumulation of its substrate, glucocerebroside, in macrophages of the liver, spleen, bones, lungs, and even the brain, forming typical storage cells known as "Gaucher cells." This accumulation causes lesions in body tissues and organs, which progressively worsen and may even lead to life-threatening bleeding.^[1]In 2018, Gaucher's disease was included in China's "First List of Rare Diseases."^[2]。

Vepure® China Launch Event - Beijing

Gaucher's disease often occurs during childhood/adolescence. The latest expert consensus on the diagnosis and treatment of Gaucher's disease in Chinese children shows that about two-thirds of1Patients with type Gaucher disease onset in childhood, and typically, the earlier the onset, the more severe the symptoms.^[3]Moreover, approximately 30%-80% of pediatric/adolescent patients may experience developmental disorders, with earlier onset leading to more significant impairment in height.^[4]。Professor Xiaoping Luo from Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology"Early diagnosis and treatment of Gaucher disease are crucial for improving the final outcome of the disease. As children with Gaucher disease age, the accumulation of substances in their bodies increases continuously, leading to more severe organ and bone damage and potentially irreversible complications, which significantly impact the physical and mental health of the child. If children with Gaucher disease can receive specific enzyme replacement therapy shortly after an early diagnosis, it will alleviate their symptoms and improve their prognosis."

Currently, enzyme replacement therapy is recommended by authoritative guidelines/consensus in China and internationally.1The standard treatment for patients with Gaucher disease is enzyme replacement therapy. By specifically supplementing the enzyme that is lacking in the patient's body, it can reduce the accumulation of glucocerebroside in the body, improve the clinical symptoms and signs of the patient, and maintain normal growth and development.^[5]. In April 2021, Takeda's Veplry^®(Injectable Velaglucerasea) Approved by the China National Medical Products Administration for use in1Long-term enzyme replacement therapy for Gaucher disease patients brings new hope for life to those with Gaucher disease.

It is reported that Vepure^®(Injectable Velaglucerasea) is a Gaucher disease enzyme replacement therapy produced in human fibroblast cell lines using gene activation technology, with an amino acid sequence identical to that of natural human glucocerebrosidase.^[5]，[6], which can improve anemia and thrombocytopenia symptoms in pediatric and adult patients, reduce the size of hepatosplenomegaly, improve related bone diseases in the early stage of treatment, and continuously improve or stabilize patient symptoms in the later stage.^[7]，[8]。Professor Lian-Sheng Zhang from the Second Hospital of Lanzhou University introduced:"Gaucher's disease is one of the few rare diseases with a clear treatment method and therapeutic effect. We are very pleased to see another related innovative therapy launched in China, bringing more treatment options to Chinese Gaucher's disease patients and helping more of them return to a normal life as soon as possible."

During the conference, Takeda announced plans to collaborate with MagnaMed Health to launch the "Care for a New Life" Patient Assistance Program. Together, they will engage in innovative payment partnerships, offering diversified payment channels and methods to alleviate the financial burden of treatment for patients and provide stronger payment support for Gaucher disease patients in China.Mr. Guohong Shan, President of Takeda ChinaTakeda, adhering to the core concept of 'patient first,' always pays close attention to the unmet treatment needs of patients with rare diseases in China. While accelerating the introduction of innovative therapies, Takeda also actively collaborates with all parties to jointly explore diagnosis, treatment, and security solutions for rare diseases such as Gaucher's disease, fully realizing patient care and building new hope for life for patients with rare diseases in China.

[1] Red Blood Cell Disease (Anemia) Group of the Hematology Branch of the Chinese Medical Association. Expert Consensus on Diagnosis and Treatment of Gaucher Disease in Chinese Adults (2020) [J]. Chinese Medical Journal, 2020, 100(24): 1841-1849.

[2] People's Health Network, Five departments including the National Health Commission jointly formulated the "First Batch of Rare Disease Catalog".http://health.people.com.cn/n1/2018/0611/c14739-30050014.html

[3] Expert Consensus on the Diagnosis and Treatment of Gaucher Disease in Chinese Children (2021)

[4] Multidisciplinary Team for Rare Diseases, Peking Union Medical College Hospital. Expert Consensus on Multidisciplinary Diagnosis and Treatment of Gaucher Disease (2020) [J]. Journal of Peking Union Medical College, 2020, 11(6): 682-697.

[5] VPRIV® (velaglucerase alfa). Summary of Product Characteristics. 2020

[6] Brumshtein B, et al. Glycobiology. 2010 Jan;20(1)24-32

[7] Ari Zimran, et al. Am J Hematol. 2015 Jul; 90(7): 577–583.

[8] Hughes DA, et al. Am J Hematol, 2015;90:584–91

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