WUXI, China, Oct. 1, 2023 /PRNewswire/ -- WuXi Biologics ('WuXi Bio') (2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO), congratulates its strategic partner, Amicus Therapeutics ('Amicus') (Nasdaq: FOLD), on receiving U.S. FDA's approval for Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules.

2023年10月1日，中国无锡/PRNewswire/-无锡生物科技（'WUXI Bio'）（2269.HK），一家领先的全球合同研究，开发和制造组织（CRDMO），祝贺其战略合作伙伴Amicus Therapeutics（'Amicus'）（纳斯达克股票代码：FOLD）获得美国FDA对Pombiliti的批准™ （cipaglucosidase alfa atga）+Opfolda™ （米格司他）65mg胶囊。

This two-component therapy is indicated for those adults living with late-onset Pompe disease (LOPD) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT)..

这种双组分疗法适用于体重≥40 kg的迟发性庞贝病（LOPD）且目前的酶替代疗法（ERT）尚未改善的成年人。。

Pombiliti™, a recombinant human GAA enzyme (rhGAA) naturally expressed with high levels of bis-M6P (Mannose 6-Phosphate), was started at WuXi Biologics in 2012 with just an initial concept and now realizes commercialization enabled by WuXi Biologics' proprietary integrated technology platform and unparalleled manufacturing capacity.

Pombiliti™, 天然表达高水平bis-M6P（甘露糖6-磷酸）的重组人GAA酶（rhGAA）于2012年在无锡生物科技有限公司启动，只是一个初步概念，现已实现无锡生物科技专有集成技术平台实现商业化和无与伦比的制造能力。

As the exclusive commercial drug substance manufacturing partner and key commercial drug product supplier, WuXi Biologics is now providing commercial manufacturing services for Pombiliti™ at five facilities across its global manufacturing network..

作为独家商业原料药制造合作伙伴和主要商业药品供应商，无锡生物制品公司目前正在为Pombiliti提供商业制造服务™ 在全球制造网络的五个设施中。。

Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, commented, 'This approval marks a major milestone for the Pompe disease community in the United States. Bringing this new treatment option to market has required immense dedication and strong partnership between the WuXi Biologics team and Amicus, relying on our shared dedication to patients and our ability to execute on our vision to bring high quality therapies to the rare disease community.

Amicus Therapeutics的总裁兼首席执行官布拉德利·坎贝尔（Bradley Campbell）评论说：“这一批准标志着美国庞贝病界的一个重要里程碑。将这种新的治疗方案推向市场需要无锡生物制剂团队与Amicus之间的巨大奉献和牢固合作，依靠我们对患者的共同奉献和我们执行我们愿景的能力，为罕见病社区带来高质量的治疗。

Over many years of hard work, our joint efforts have brought this medicine from an idea, to a molecule, to clinical manufacturing and commercial supply, delivering on our commitment to people living with late-onset Pompe disease.'.

经过多年的努力，我们的共同努力使这种药物从一个想法，一个分子，到临床制造和商业供应，实现了我们对迟发性庞贝病患者的承诺。

Dr. Chris Chen, CEO of WuXi Biologics, commented, 'We're honored to enable Amicus to achieve this great milestone, which we've been looking forward to for over 11 years. This close partnership not only showcases our successful strategy, 'follow the molecule', but also inspires us to accelerate the discovery, development and manufacturing of more rare disease biologic therapeutics for global partners.

无锡生物科技首席执行官Chris Chen博士评论说：“我们很荣幸能够使Amicus实现这一伟大的里程碑，我们一直期待超过11年。这种密切的合作关系不仅展示了我们成功的战略“遵循分子”，而且激励我们加速为全球合作伙伴发现，开发和制造更罕见的疾病生物疗法。

I am confident that the collaboration between our two companies, both of which share a passion for patients, will achieve greater successes in the journey of benefiting global patients with rare diseases.'.

我相信，我们两家公司之间的合作，这两家公司都对患者充满热情，将在使全球罕见病患者受益的旅程中取得更大的成功。

About Pompe Disease

关于庞贝氏症

Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. Pompe disease ranges from a rapidly deteriorating infantile form with significant impact to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle and progressive respiratory involvement.

庞贝病是由酸性α-葡萄糖苷酶（GAA）缺乏引起的遗传性溶酶体疾病。GAA水平降低或不存在会导致糖原在细胞中积累，这被认为会导致庞贝病的临床表现。庞贝病的范围从对心脏功能有重大影响的快速恶化的婴儿形式到主要影响骨骼肌和进行性呼吸系统受累的更缓慢的进行性，迟发性形式。

Late-onset Pompe disease can be severe and debilitating with progressive muscle weakness throughout the body that worsens over time, particularly skeletal muscles and muscles that control breathing..

迟发性庞贝氏症可能是严重的和衰弱的，随着时间的推移，全身肌肉无力逐渐恶化，特别是骨骼肌和控制呼吸的肌肉。。

About Pombiliti + Opfolda

关于Pombiliti+Opfold

Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that's designed to reduce loss of enzyme activity in the blood..

Pombiliti+Opfolda是一种双组分疗法，由cipaglucosidase alfa atga组成，这是一种双M6P富集的rhGAA，能够促进通过M6P受体的高亲和力摄取，同时保持其加工成最活跃形式的酶的能力，以及口服酶稳定剂miglustat，旨在减少血液中酶活性的损失。。

About WuXi Biologics

关于无锡生物制品

WuXi Biologics (stock code: 2269.HK) is a leading global Contract Research, Development and Manufacturing Organization (CRDMO) offering end-to-end solutions that enable partners to discover, develop and manufacture biologics – from concept to commercialization – for the benefit of patients worldwide..

无锡生物制品（库存代码：2269.HK）是一家领先的全球合同研究，开发和制造组织（CRDMO），提供端到端解决方案，使合作伙伴能够发现，开发和制造生物制品-从概念到商业化-全球患者受益。。

With over 12,000 skilled employees in China, the United States, Ireland, Germany and Singapore, WuXi Biologics leverages its technologies and expertise to provide customers with efficient and cost-effective biologics discovery, development and manufacturing solutions. As of June 30, 2023, WuXi Biologics is supporting 621 integrated client projects, including 22 in commercial manufacturing..

无锡生物科技利用其技术和专业知识，为客户提供高效，经济的生物制剂发现，开发和制造解决方案，拥有12000多名技术人员。截至2023年6月30日，无锡生物科技正在支持621个综合客户项目，其中商业制造业22个项目。。

WuXi Biologics views Environmental, Social, and Governance (ESG) responsibilities as an integral component of our ethos and business strategy, and we aim to become an ESG leader in the biologics CRDMO sector. Our facilities use next-generation biomanufacturing technologies and clean-energy sources. We have also established an ESG committee led by our CEO to steer the comprehensive ESG strategy and its implementation, enhancing our commitment to sustainability..

无锡生物科技将环境，社会和治理（ESG）责任视为我们精神和业务战略的组成部分，我们的目标是成为生物制品CRDMO部门的ESG领导者。我们的设施使用下一代生物制造技术和清洁能源。我们还成立了由首席执行官领导的ESG委员会，以指导全面的ESG战略及其实施，加强我们对可持续发展的承诺。。

For more information about WuXi Biologics, please visit: www.wuxibiologics.com.

有关无锡生物制剂的更多信息，请访问：www.wuxibiologics.com。

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SOURCE WuXi Biologics

来源无锡生物制品