Pfizer's Innovative Rare Disease Drug Vyndamax® (Tafamidis) Debuts at China International Import Expo

Shanghai, November 6, 2020 /PRNewswire/ -- At the third China International Import Expo, Pfizer's innovative rare disease drug tafamidis soft capsules (Tafamidis, brand name: Vyndaqel)^® ，VYNDAMAX ^®) was launched with great fanfare. Tafamidis meglumine soft capsules, which were officially approved in China at the end of September, are the first and only drug approved globally for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Their introduction fills the previous gap in China where there were no effective drugs for ATTR-CM treatment, providing new options for ATTR-CM patients to better manage their condition and delay disease progression. On October 30, Vyndaqel^®Won the Prix Galien Awards for Best Biotechnology Product.

Li Linkang, Chairman of the Board, Deputy Director of the Office of the National Rare Disease Diagnosis and Treatment Collaboration Network, Executive Chairman of the Chinese Alliance for Rare Diseases, and President of the Beijing Society for Rare Disease Diagnosis, Treatment, and Security; Professor Tian Zhuang, Associate Chief Physician in the Department of Cardiology and Deputy Director of the International Medical Services Department at Peking Union Medical College Hospital; Professor Huo Li, Director of the Department of Nuclear Medicine at Peking Union Medical College Hospital; Mr. Huang Rufang, Founder and Director of the Cord Rare Disease Center; Mr. Wu Kun, Chief Operating Officer of Pfizer Biopharmaceuticals Group China; and Ms. Wang Yiqin, Vice President of Marketing for Pfizer Biopharmaceuticals Group China, were among the distinguished guests who attended and witnessed this historic moment. The attendees shared their perspectives on the diagnostic and therapeutic challenges of ATTR-CM, as well as the overall development planning for the diagnosis and treatment of ATTR-CM and rare diseases. They also engaged in in-depth discussions on topics such as collaborating to elevate the standard of rare disease diagnosis and treatment in China and improving the current state of care for patients with rare diseases in the country.

Group Photo at the Launch Ceremony of Vyvanse® (from left to right: Ms. Wang Yiqin, Vice President of Marketing, Pfizer Biopharmaceuticals Group China; Ms. Li Jinhui, General Manager of Oncology and Rare Diseases, Pfizer Biopharmaceuticals Group China; Mr. Wu Kun, Chief Operating Officer, Pfizer Biopharmaceuticals Group China; Mr. Huang Rufang, Founder and Director of the Cord Center for Rare Diseases; Chairman Li Linkang, Deputy Director of the Office of the National Collaboration Network for Diagnosis and Treatment of Rare Diseases, Executive Chairman of the Chinese Alliance for Rare Diseases, and President of the Beijing Society for Diagnosis and Treatment and Security of Rare Diseases; Dr. Cao Junyang, Vice President and Chief Medical Officer, Pfizer Biopharmaceuticals China; Ms. Shi Danni, Head of Rare Disease Medical Affairs, Pfizer Biopharmaceuticals Group China)

Integrate High-Quality Resources to Jointly Promote the Development of Rare Disease Prevention and Control in China

Since the 18th National Congress of the Communist Party of China, the CPC Central Committee and the State Council have adhered to the people-centered development philosophy, attaching great importance to the management of rare diseases. Relevant departments, including the National Health Commission, the National Medical Products Administration, and the National Healthcare Security Administration, have implemented a series of measures to enhance diagnostic and therapeutic capabilities for rare diseases, optimize the review and approval processes for pharmaceuticals, and improve drug accessibility, thereby promoting the development of rare disease healthcare in China.

“The Chinese Rare Disease Alliance remains committed to a patient-centered approach, dedicated to integrating high-quality resources from all sectors—including clinical diagnosis and treatment, medical insurance, pharmaceutical companies, and social welfare organizations—to help enhance the standards of rare disease diagnosis, treatment, and coverage in China, thereby promoting overall progress in clinical care, drug development, and disease management for rare diseases,” stated Chairman Li Linkang. “Taking ATTR-CM as an example, with the launch of innovative therapies, patients are no longer left with ‘no treatment options.’ In the future, we will continue to consolidate high-quality resources and devote substantial efforts to improving disease diagnosis and treatment, enhancing drug accessibility, and strengthening medical security, thereby helping ATTR-CM patients improve their quality of life and ensuring that individuals with rare diseases no longer feel isolated or unsupported.””

Joining Forces, Collaborating for ProgressEstablishment of an Innovative Model for the Diagnosis and Treatment of ATTR-CM

Due to the low awareness of ATTR-CM, clinical diagnosis and treatment currently face numerous severe challenges. Only a few hospitals in China possess the capability for diagnosis and treatment, which is far from meeting patient needs; therefore, it is urgent to establish a comprehensive and systematic disease management framework. Since 2019, under the leadership of the Rare Disease Alliance, Centers of Excellence (CoEs) for ATTR-CM diagnosis and treatment have been successively established in China, aiming to promote the improvement and standardization of diagnostic and therapeutic capabilities, gradually forming a standardized care model. Professor Tian Zhuang pointed out, “We have successfully established the first batch of leading demonstration centers and launched a series of educational initiatives extending to lower-tier medical consortium institutions, exploring issues related to two-way referrals and patient management during the diagnostic and treatment process. It is expected that within the next 2–3 years, through continuous refinement of the diagnostic and treatment system, a standardized ATTR-CM care framework will cover most provinces and regions in China. At that time, we hope to provide patients with more convenient and efficient diagnostic and treatment pathways, actively striving to save time for treatment.”

ATTR-CM is a multisystem disease, with patients often scattered across various departments such as neurology, ophthalmology, and cardiology. Due to the atypical symptoms at onset, patients are frequently misdiagnosed or experience delayed treatment. Therefore, promoting multidisciplinary team (MDT) diagnosis and treatment within hospitals is of great significance for early diagnosis and targeted therapy of the disease.

“For patients with ATTR-CM, the establishment of multidisciplinary team (MDT) services and remote consultations has made timely diagnosis and standardized treatment possible, thereby effectively reducing misdiagnosis and inappropriate treatment. This approach not only minimizes the burden of seeking medical care across multiple facilities but also alleviates patients’ financial burden to a certain extent,” pointed out Professor Huo Li.““Grounded in the standardized diagnosis and treatment of diseases, the Rare Disease Alliance collaborates with relevant departments to facilitate the establishment of multidisciplinary team (MDT) structures across hospitals at all levels within the healthcare system. This initiative aims to accelerate the enhancement of MDT capabilities, thereby improving diagnostic and therapeutic proficiency for these conditions, increasing the precision and efficiency of patient diagnoses, and progressively achieving the goal of early diagnosis and early treatment. By promoting the construction of Centers of Excellence (CoE) diagnostic and treatment systems and enhancing in-hospital MDT capabilities, we are striving to explore a replicable and referenceable single-disease management model for rare diseases. This effort will further advance the development of rare disease healthcare in China, benefiting a greater number of rare disease patients across the country,” stated Chairman Li Linkang.

“A” Promising Future, A Renewed “Heart”: Pfizer Continues to Break Through in Improving Patient Diagnosis and Treatment

Rare diseases have long posed a challenge to humanity as a whole. Compared with other diseases, significant gaps exist in rare disease management, ranging from disease awareness, diagnosis, and treatment to drug accessibility and affordability for patients. With the market launch of innovative therapeutic agents, patients in China with ATTR-CM can now achieve better disease remission and improved quality of life. Furthermore, enhancing patient awareness of ATTR-CM will serve as an effective means to improve disease diagnosis and treatment rates.

Mr. Huang Rufang stated, “As a patient organization focused on the field of rare diseases, we are also actively engaged in patient education and disease awareness initiatives to enhance public and patient understanding of these conditions, enabling more patients to achieve early diagnosis and receive better treatment through proactive screening or diagnostic evaluation. Meanwhile, our extensive experience accumulated over the years will further promote patient management and communication efforts specifically for ATTR-CM as a single-disease entity. Furthermore, given the unique circumstances of patients with rare diseases, I hope that society as a whole will extend greater attention and care to them.”

“‘Delivering breakthrough innovations that change patients’ lives’ is Pfizer’s enduring commitment. We have always centered our efforts on the needs of patients with rare diseases, accelerating the introduction of innovative, breakthrough therapies to improve treatment accessibility and ensure that more patients have access to effective medications,” said Mr. Wu Kun. “In response to the current landscape of diagnosis and treatment for rare diseases, particularly ATTR-CM, we will continue to collaborate with alliances, patient organizations, and other partners to advance the standardization of rare disease care and strengthen healthcare systems across China, thereby delivering greater benefits to patients and contributing to the early realization of ‘Healthy China 2030.’”