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Classified in: Health
Subject: FDA

WuXi Biologics Congratulates Amicus on Receiving FDA Breakthrough Therapy Designation for AT-GAA

- The second product successfully receiving Breakthrough Therapy Designation that WuXi Biologics has enabled its partner to develop

SHANGHAI, Feb. 27, 2019 /PRNewswire/ -- 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) on receiving FDA Breakthrough Therapy Designation for AT-GAA (ATB200/AT2221) in late onset Pompe disease, an inherited lysosomal storage disorder caused by the deficiency of an enzyme known as acid alpha-glucosidase (GAA). AT-GAA is the first ever investigational product for Pompe disease to receive breakthrough designation.  

The AT-GAA program was initiated at WuXi Biologics in 2012 with just an initial concept and now progresses through a pivotal study enabled by the global leading technology platform and unparalleled manufacturing capacity at WuXi Biologics. Just this month the two companies signed an exclusive manufacturing partnership, through which WuXi Biologics will provide dual commercial sources for both drug substance and drug product of ATB200 and will be the exclusive commercial drug substance partner and key commercial drug product supplier. 

"We congratulate Amicus on achieving this exciting milestone," said Dr. Chris Chen, CEO of WuXi Biologics. "It is a great honor to enable global innovative partners like Amicus through our world-class development and manufacturing capacities and capabilities. We are confident of supplying this critical product to the global market and wish the program a great success to benefit patients worldwide."

This is the second product successfully receiving Breakthrough Therapy Designation that WuXi Biologics has enabled its partner to develop. TrogarzoTM, the first to do so, has been approved by the U.S. FDA in March 2018, marking the first commercial product manufactured by WuXi Biologics for the U.S. market.

About AT-GAA

AT-GAA is an investigational therapy that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In preclinical studies, AT-GAA was associated with increased tissue enzyme levels, reduced glycogen levels in muscle, and improvements in muscle strength. A global Phase 1/2 study (ATB200-02) is ongoing to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of AT-GAA.

Amicus has also initiated PROPEL, a global Phase 3 clinical study (also known as ATB200-03) of AT-GAA in adult patients with late onset Pompe disease. PROPEL is a 52-week, double-blind randomized study designed to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa, an enzyme replacement therapy (ERT). More information, including a list of participating sites, is available at www.clinicaltrials.gov: NCT03729362.

About Amicus

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 on Twitter and LinkedIn.

About WuXi Biologics

WuXi Biologics (stock code: 2269.HK), a Hong Kong-listed company, is the only open-access biologics technology platform in the world offering end-to-end solutions to empower organizations to discover, develop and manufacture biologics from concept to commercial manufacturing. Our company history and achievements demonstrate our commitment to providing a truly ONE-stop service offering and value proposition to our global clients. As of June 30, 2018, there were a total of 187 integrated projects, including 98 projects in pre-clinical development stage, 78 projects in early-phase (phase I and II) clinical development, 10 projects in late-phase (phase III) development and 1 project in commercial manufacturing. With total estimated capacity of biopharmaceutical production planned in China, Ireland, Singapore and US reaching 220,000 liters by 2021, we will provide our biomanufacturing partners with a robust and premier-quality global supply chain network. For more information on WuXi Biologics, please visit www.wuxibiologics.com.

SOURCE WuXi Biologics

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