Replagal® (Agalsidase Alfa Concentrated Solution for Infusion) Receives First Prescription in China at Shandong Provincial Hospital

JINAN, May 18, 2021 /PR Newswire/ -- Today, Replagal, a Fabry disease treatment drug introduced by Takeda China,^®(Agalsidase alfa concentrated solution for injection) issued the first prescription in China at Shandong Provincial Hospital Affiliated to Shandong First Medical University, bringing a novel clinical treatment option for Chinese patients with Fabry disease (alpha-galactosidase A deficiency) and helping them achieve a wonderful life with enzymes. As an enzyme replacement therapy derived from and manufactured in human cells, Replagal^®(Agalsidase alfa concentrate for solution for injection) can provide long-term cardiac and renal protection and slow disease progression.^[1], improving the quality of life for patients and their families. Replagal^®(Agalsidase alfa Concentrated Solution for Injection) was approved by the National Medical Products Administration in August 2020 for the treatment of patients diagnosed with Fabry disease.

Professor Wang Rong, Vice President of Shandong Provincial Hospital Affiliated to Shandong First Medical University, issued China's first prescription for Ruipujia®.

Fabry disease is a rareX-linkedA hereditary lysosomal storage disorder, this condition occurs when the metabolite globotriaosylceramide (GL-3) cannot be properly cleaved, leading to its extensive accumulation in various tissues and organs. Patients typically experience severe burning pain in the hands and feet, accompanied by hypohidrosis, anhidrosis, and impaired thermoregulation. As the disease progresses, symptoms gradually worsen, resulting in renal and cardiac complications such as severe renal failure or even uremia, as well as cerebrovascular and cardiovascular events like stroke and myocardial infarction. Without effective treatment, the condition is life-threatening. In 2018, Fabry disease was included in the *First Batch of Rare Diseases Catalog*, jointly formulated by five departments including the National Health Commission.^[2]。

Professor Wang Rong, Vice President of Shandong Provincial Hospital Affiliated to Shandong First Medical University, stated: "In the past, the lack of disease-specific therapies for Fabry disease in China meant that most patients were unable to control disease progression and had to rely solely on symptomatic management, such as analgesics for severe pain episodes or dialysis for severe renal impairment. Fabry disease placed a heavy burden on patients and their families. Fortunately, in recent years, rare diseases have garnered increasing attention from all sectors. Supported by a series of favorable national policies, novel treatment regimens for Fabry disease have been rapidly introduced into China, bringing new hope for improving patients' survival rates and quality of life."

Dr. Yu Che from the Department of Nephrology, Provincial Hospital Affiliated to Shandong First Medical University, stated: “Enzyme replacement therapy (ERT) is the standard treatment for Fabry disease recommended by authoritative domestic and international guidelines and consensus statements. Standardized, adequate-dose, early ERT plays a crucial role in improving clinical outcomes and preventing severe complications in patients with Fabry disease. Today, the first prescription in China for a new enzyme replacement therapy was issued at our hospital, which will provide patients with a powerful new tool to combat Fabry disease, empowering them with greater courage and confidence to look forward to a better life.”

To further enhance patient access to innovative drugs for rare diseases, Shandong Province has actively explored innovative models in recent years to advance the coverage of critical illness medical insurance. With particular attention to rare disease patients who have special treatment needs, the province has continuously improved the medication security mechanism for this population. On December 14, 2020, the Shandong Provincial Healthcare Security Administration issued the 《Notice on Further Improving the Provincial Critical Illness Insurance System》.^[3]，the inclusion of specialty drugs with proven clinical efficacy for three rare diseases, including Fabry disease, into the medical insurance reimbursement scope has significantly alleviated the financial burden of treatment for Fabry disease patients in Shandong Province, making the hope of accessing highly effective treatment regimens no longer a distant dream.

[1] Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: a 10-year Fabry Outcome Survey (FOS) analysis. Ramaswami U, et al. Drug Des Devel Ther. 2019:13:3705-3715.

[2] People's Health Network, the National Health Commission and four other departments jointly formulated the 《Catalog of Rare Diseases (First Batch)》,http://health.people.com.cn/n1/2018/0611/c14739-30050014.html

[3] http://www.qingdao.gov.cn/zwgk/xxgk/ybj/gkml/gwfg/202012/t20201231_2877380.shtml

Statement:

1. This article aims to disseminate cutting-edge medical and pharmaceutical information and does not constitute a recommendation or promotion of any drug or diagnostic and treatment regimen.
2. For more information on diseases, medications, or diagnosis and treatment, please consult a healthcare professional.

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