中国，上海
2021年10月9日
全球领先的开放式生物制药技术平台公司药明生物（WuXi Biologics, 2269.HK）祝贺战略合作伙伴Amicus（纳斯达克股票代码：FOLD）新一代庞贝病疗法AT-GAA（ATB200/AT2221）上市申请获得美国FDA受理。AT-GAA由重组人酸α-葡糖苷酶（rhGAA）ATB200和麦格司他（miglustat）AT2221组成，FDA已分别受理两者的生物制剂上市许可申请（BLA）和新药上市申请（NDA）。
AT-GAA于2012年在药明生物平台上从一个“主意”诞生。在过去的十年中，Amicus与药明生物密切合作，将该“主意”转化为具有临床疗效的新药，并推动其成为庞贝病下一代标准疗法。Amicus公司董事长兼首席执行官John F. Crowley曾描述开发该产品稳定且高质量的大规模生产工艺的难度类似于“研发航天飞机”，并对药明生物的技术能力和质量体系表示了高度认可。2019年2月，药明生物与Amicus建立战略合作，成为ATB200项目商业化阶段的独家原液生产合作伙伴和主要制剂供应合作伙伴。
Amicus公司董事长兼首席执行官John F. Crowley先生表示：“此次FDA受理AT-GAA的上市申请为美国庞贝病患者带来了福音，而这一成绩离不开众多人和团队的辛勤努力，药明生物团队在其中更是做出了重要贡献。他们以极大的热情、丰富的专业知识以及无私的敬业精神，为高质量产品保驾护航。我们对于双方的紧密合作关系感到非常自豪，也期待未来继续携手加快AT-GAA在全球的上市进程，惠及广大患者。”
“我们衷心祝贺Amicus取得这一重要里程碑，”药明生物首席执行官陈智胜博士表示，“我们很荣幸通过药明生物领先的技术平台和强劲的产能赋能Amicus开发新一代庞贝病疗法ATB200，这也是药明生物首个应用连续收获的创新灌流细胞培养工艺的BLA项目。该项目BLA获得FDA受理也充分证明了我们坚持赋能全球合作伙伴，造福全球庞贝病患者的决心。”
关于AT-GAA
AT-GAA（ATB200/AT2221）是由重组人酸α-葡糖苷酶（rhGAA）和麦格司他（miglustat）联用的组合疗法。ATB200是一种独特的重组人酸α-葡糖苷酶（rhGAA），能够优化碳水化合物结构，特别是甘露糖-6磷酸盐（M6P）。AT2221是α-葡糖苷酶稳定剂麦格司他（miglustat）。临床前研究显示，AT-GAA与成熟GAA溶酶体水平增加、肌肉中糖原水平降低、自噬缺陷缓解及肌肉力量改善有关。
关于庞贝病
庞贝病是一种由酸性α-葡萄糖苷酶（GAA）缺乏引起的遗传性溶酶体疾病，临床表现为GAA水平的降低或缺失并导致细胞中糖原的积累。庞贝病可能会使人虚弱，以严重的肌肉无力为特征，并随着时间的推移而恶化。庞贝病涵盖婴儿型到晚发型，前者对心脏功能有显著影响，疾病迅速进展至死亡，后者主要影响骨骼肌，疾病进展相对缓慢。据估计，全球庞贝病患者约5000至10000人。
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关于Amicus Therapeutics
Amicus Therapeutics（纳斯达克股票代码：FOLD）是一家致力于为罕见代谢疾病患者发现、开发和提供高质量创新药的全球性生物技术公司。公司立足于“以患者为中心”，针对罕见代谢疾病打造了强大的先进在研产品线。更多信息请访问www.amicusrx.com，以及Twitter和LinkedIn。
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WuXi Biologics Congratulates Amicus on U.S. FDA's Acceptance of its Filing for AT-GAA for the Treatment of Pompe Disease
SHANGHAI
October 9, 2021
WuXi Biologics (“WuXi Bio”) (Stock code: 2269.HK), a leading global open-access biologics technology platform company offering end-to-end solutions for biologics discovery, development and manufacturing, congratulates its strategic partner Amicus Therapeutics (“Amicus”) (Nasdaq: FOLD) that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat for AT-GAA (ATB200/AT2221), Amicus' investigational two-component therapy for the treatment of Pompe disease.
The AT-GAA program was mostly a concept when it was initiated at WuXi Biologics in 2012. Over the last 10 years, WuXi Biologics has closely worked with Amicus to enable this idea and potential of this medicine to become the next standard of care for the treatment of Pompe disease. In February 2019, WuXi Biologics and Amicus announced a strategic collaboration for ATB200 under which WuXi Biologics will be the exclusive commercial drug substance manufacturing partner and key commercial drug product supplier.
John F. Crowley, Chairman and CEO of Amicus Therapeutics, commented, “This is a major step forward for people living with Pompe disease in the United States. Amicus is committed to bringing AT-GAA to all persons living in every part of the world, as fast as possible. To do so requires the efforts of many people and teams. At the center of our efforts and success to date has been the WuXi Biologics team that has worked tirelessly with great passion and expertise to ensure that we manufacture the highest quality biologic. I am immensely proud of this relationship and it will continue for many years ahead.”
“We congratulate Amicus on this important milestone,” said Dr. Chris Chen, CEO of WuXi Biologics. “We are proud to support Amicus on the development and manufacturing of ATB200 through our proprietary technology platforms and robust capabilities. It's the first BLA program applying WuXi Biologics' novel perfusion cell culture manufacturing technology with continuous product harvest. It also serves as a testament to our determination and persistence to enable our partners to develop breakthrough therapy for the treatment of Pompe disease.”
About AT-GAA
AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to enhance uptake into cells, administered in conjunction with miglustat (AT2221), a stabilizer of cipaglucosidase alfa. In preclinical studies, AT-GAA was associated with increased levels of the mature lysosomal form of GAA and reduced glycogen levels in muscle, alleviation of the autophagic defect and improvements in muscle strength.
About Pompe Disease
Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA levels lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company’s website at www.amicusrx.com, and follow us on Twitter and LinkedIn.
About WuXi Biologics
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