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.”
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
WuXi Biologics (stock code: 2269.HK), a Hong Kong-listed company, is a leading global open-access biologics technology platform offering end-to-end solutions to empower organizations to discover, develop, and manufacture biologics from concept to commercial manufacturing. The company’s history and achievements demonstrate its commitment to providing a truly ONE-stop service offering and strong value proposition to its global clients.
The company is currently conducting on behalf of its clients and partners (as of June 30, 2021) a total of 408 integrated projects, including 212 in pre-clinical development stage, 160 in early-phase (phase I and II) clinical development, 32 in late-phase (phase III) development and 4 in commercial manufacturing. With a total estimated capacity of exceeding 430,000 liters for biopharmaceutical production planned after 2024 in China, Ireland, the U.S., Germany, and Singapore, WuXi Biologics will provide its biomanufacturing partners with a robust and premier-quality global supply chain network.
WuXi Biologics views Environmental, Social, and Governance (ESG) responsibilities as an integral component of its ethos and business strategy and aims to become a global ESG leader in biologics manufacturing. We use next-generation clean biomanufacturing technologies and utilize cleaner energy sources. We have also established an ESG committee led by the CEO to increase efficiency while advancing commitment to sustainability. For more information about WuXi Biologics, please visit: www.wuxibiologics.com.