[Amicus Therapeutics Co., Ltd.] Expanded clinical trial for Pompe disease started - CIPAGLUCOSIDASE ALFA and Miglustat combined administration -

Amicus Therapeutics Co., Ltd. Amicus Therapeutics Co., Ltd. (Headquarters: Chiyoda-ku, Tokyo; Representative Director: Simon Collins) is pleased to announce that adult late-onset Pompe disease patients in Japan We are pleased to inform you that we have started an expanded clinical trial of cipaglucosidase alfa / miglustat, a new combination therapy under development, targeting Two formulations of enzyme replacement therapy are currently approved in Japan, but patients and caregivers are eager for new treatment options to become available. Overseas, Amicus Therapeutics Inc. has implemented multiple Expanded Access Programs in parallel with company-led clinical trials to meet the needs of Pompe disease patients. Similarly, in Japan, we have decided to start an expanded clinical trial to meet the needs of patients. This is an expansion trial with a primary focus on safety, with no control group, and all subjects will receive biweekly cipaglucosidase alfa/miglustat combination therapy. Details of this clinical trial can be viewed from the following external site: jRCT (clinical research implementation plan and research summary publication system). https://jrct.niph.go.jp/latest-detail/jRCT2061210056Investigational drug cipaglucosidase alfa/miglustat A novel combination therapy for the treatment of Pompe disease. Cipaglucosidase alfa is a recombinant human acid α-glucosidase with increased mannose-6-phosphate (M6P) for improved cellular uptake of the enzyme. It is expected to reduce glycogen, a major storage substance in Pompe disease, especially in muscle tissue. Cipaglucosidase alfa is administered by intravenous infusion every other week. Miglustat (N-butyl-deoxynojirimycin) is an oral solid formulation that is expected to stabilize cipaglucosidase alfa in the blood and increase the transport of many enzymes that retain their activity to target tissues. I'm here. It is administered 1 hour before the start of intravenous infusion of cipaglucosidase alfa. Development status of this product In Japan, Study ATB200-03 and its long-term extension study (Study ATB200-07) are being conducted as the main clinical trial in adult Pompe disease patients. increase. In December 2020, it was designated as an orphan drug by the Ministry of Health, Labor and Welfare (MHLW). Overseas, it was designated as a breakthrough therapy by the U.S. Food and Drug Administration (FDA) in February 2019, and an application for approval has already been submitted. A regulatory application has also been filed with the European Medicines Agency (EMA). In January 2020, it was designated as a Promising Innovative Medicine (PIM) by the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom. About Pompe disease (Glycogen storage disease type II)A rare common disease caused by mutations in the gene encoding acid alpha-glucosidase (GAA), an enzyme involved in glycogen breakdown within lysosomes. It is a chromosomal recessive (recessive) disease. Dysfunction of this enzyme leads to accumulation of glycogen in lysosomes, progressive impairment of cell function, and progressive decline in muscle strength, locomotion and lung function. An estimated 5,000 to 10,000 people worldwide have Pompe disease. For symptoms, please refer to "Pompe Note", a document that describes Pompe disease in an easy-to-understand manner: For those involved with children with Pompe disease. https://amicusrx.jp/ge/pompe.htmlAmicus Therapeutics Inc.Patient-centric, US-based biotechnology company offering new therapies for patients with rare metabolic diseases We are a global company engaged in the discovery, development and marketing of pharmaceuticals to deliver high quality therapeutics. Above all, we are dedicated to advancing and expanding our pipeline of cutting-edge breakthrough medicines that are novel and useful in metabolic diseases, for patients. For more information, please visit the following sites. http://www.amicusrx.comAmicus Therapeutics Inc.The Japanese subsidiary of Amicus Therapeutics Inc. For Fabry disease, a type of lysosomal storage disease similar to Pompe disease, the company has a track record of in-house clinical development and acquisition of manufacturing and marketing approval. We are selling. We will continue to contribute to medical care by working to eliminate unapproved drugs and off-label drugs that are in high medical need. http://www.amicusrx.jp or moreNP-AT-JP-00011221

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