First National Prescription for Vivenxin®, an Innovative Drug for Rare Diseases

December 7, 2020 – Today, Vyvanxin® (chlorobenzolic acid softgel, Vyndamax®, 61 mg), an innovative drug for rare diseases recently approved in China, was prescribed at Peking Union Medical College Hospital by Dr. Tian Zhuang of the Department of Cardiology for an elderly patient with wild-type transthyretin amyloidosis cardiomyopathy The first prescription in China was written by Dr. Tian Zhuang of the Department of Cardiology at Peking Union Medical College Hospital for an elderly patient with wild-type transthyretin amyloid cardiomyopathy. Vivaxin is the world’s first and only approved oral drug for the treatment of ATTR-CM, filling the gap of no effective drug in the treatment of ATTR-CM in China, providing an innovative option for the clinical treatment of the disease and bringing new hope for the treatment of patients.

Dr. Tian Zhuang, Department of Cardiology, Peking Union Medical College Hospital

ATTR-CM is a serious threat to patients’ lives, chlorobenzolic acid softgels fill the drug gap

ATTR-CM is a rare and fatal disease with a diagnosis rate of less than 1% [1]. Its pathogenesis is based on the abnormal dissociation and misfolding of unstable transthyretin (TTR) protein, which forms amyloid-like material deposited in the myocardial interstitium, leading to restrictive cardiomyopathy and progressive heart failure in the long term.

The disease is easily confused with heart failure, and because of the lack of disease awareness, patients often miss the best treatment Time due to misdiagnosis, underdiagnosis and delayed diagnosis and treatment. In recent years, the disease has been diagnosed in hospitals in different regions of China, and the quality of Life of patients has been severely affected, with an average survival time of 2-6 years after clinical diagnosis [2],” said Professor Zhang Leyang, President of Peking Union Medical College Hospital and Vice Chairman of the China Rare Disease Alliance. The marketing and prescription of innovative drugs has changed the previous dilemma of having no Medicine for ATTR-CM, bringing hope for patients to prolong their lives.

Peking Union Medical College Hospital has issued the first prescription in China

In partnership with the Rare Disease Alliance, Peking Union Medical College (PUMC) is helping to improve the quality of life of ATTR-CM patients.

ATTR is a multi-system disease with non-specific symptoms, which often requires the collaboration of different departments to confirm the diagnosis, highlighting the importance of improving multidisciplinary treatment (MDT).

Peking Union Medical College Hospital is actively working with the China Rare Disease Consortium to promote MDT consultation within the hospital. Our rare disease team conducts MDT consultations for patients with various rare diseases every week, hoping to transform the previous ‘one-man’ treatment into a ‘collaborative’ treatment model, so that patients can receive more comprehensive and thoughtful treatment by integrating all aspects of the patient’s condition. Prof. Zhang Leyang said, “Peking Union Medical College Hospital is actively playing a leading role in demonstrating the ATTR-CM nuclear diagnosis system, and the director of the Department of Nuclear Medicine, Huo Li, has established and promoted the ATTR-CM nuclear diagnosis system in China. It is planned that in the next 2-3 years, the ATTR-CM standardized diagnosis and treatment system can cover most provinces and regions in China.


[1] Tian Zhuang, Zhang Leyang. Treatment of transthyretin amyloid cardiomyopathy. Chinese Journal of Cardiovascular diseases. 2020;48(1):86-9

[2] Maurer MS, Grogan DR, Judge DP, et al. Tafamidis in transthyretin amyloid cardiomyopathy: effects on transthyretin stabilization and clinical outcomes. Circ Heart Fail. 2015;8(3):519⁃26.

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