WuXi Biologics Congratulates Amicus Therapeutics on European Commission Approval for Pombiliti™ in Patients with Late-Onset Pompe Disease

Rhea-AI Summary

WuXi Biologics has announced its partnership with Amicus Therapeutics in celebrating the European Commission's approval of Pombiliti™ (cipaglucosidase alfa) for adults with late-onset Pompe disease. This therapy, a combined treatment with miglustat, is a significant milestone achieved through WuXi's advanced manufacturing capabilities since the therapy's inception in 2012. The approval is based on clinical data from the Phase 3 PROPEL trial, which included a diverse patient population. WuXi Biologics continues to support commercialization through its global sourcing strategy, reinforcing its position as a leader in biologics development.

Positive

• European Commission approved Pombiliti™ for late-onset Pompe disease, enhancing market position for Amicus and WuXi.
• Approval based on Phase 3 PROPEL trial data, indicating strong clinical efficacy.
• WuXi's involvement in the therapy's development since 2012 highlights their manufacturing capabilities.

Negative

• None.

WUXI, China, March 28, 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 European Commission's approval for Pombiliti™ (cipaglucosidase alfa), a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD).

Pombiliti™ 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. Since 2019, WuXi Biologics has been providing commercial manufacturing services through "Global Dual Sourcing" strategy as the exclusive commercial drug substance manufacturing partner and key commercial drug product supplier.

 "The joint efforts between Amicus and WuXi Biologics over past 11 years are just for this moment! We're honored to enable Amicus to achieve this great milestone, which will bring this new therapy to the patients with Pompe disease," commented Dr. Chris Chen, CEO of WuXi Biologics, "This provides another strong validation of our 'Follow and Win the Molecule' strategy that realizes our partners' ideas into commercialized medical treatments. Leveraging our world-class capabilities and robust global network, WuXi Biologics is accelerating and transforming how biologics are discovered, developed and manufactured globally to benefit patients worldwide."

The EC based the approval of Pombiliti™ on clinical data from the Phase 3 pivotal study (PROPEL), the only randomized, controlled trial in LOPD to include patients in the high unmet need ERT-experienced population, in addition to ERT-naïve patients. The EC approval of Pombiliti™ follows the positive opinion previously granted by the Committee for Medicinal Products for Human Use (CHMP).

About AT-GAA

AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa, a bis-M6P-enriched rhGAA which 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 minimize loss of enzyme activity in the blood. In clinical studies, AT-GAA was associated with demonstrated improvements in both musculoskeletal and respiratory measures.

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 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 fatal infantile form with significant impacts to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle and progressive respiratory involvement. Late-onset Pompe disease can be severe and debilitating, including progressive muscle weakness throughout the body, particularly the skeletal muscles and muscles controlling breathing, that worsens over time.

About WuXi Biologics

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.

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 Dec 31, 2022, WuXi Biologics is supporting 588 integrated client projects, including 17 in commercial manufacturing.

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.

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

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SOURCE WuXi Biologics

FAQ

What is the significance of the European Commission approval for Pombiliti?

The European Commission approved Pombiliti™ for adults with late-onset Pompe disease, marking a significant advancement in treatment options.

When did WuXi Biologics start working on Pombiliti?

WuXi Biologics began its work on Pombiliti™ in 2012, contributing to the therapy's development and commercialization.

What is the relationship between WuXi Biologics and Amicus Therapeutics regarding Pombiliti?

WuXi Biologics partnered with Amicus Therapeutics to develop and manufacture Pombiliti™, supporting its approval and market launch.

What is the Phase 3 PROPEL trial related to Pombiliti?

The Phase 3 PROPEL trial is a pivotal study that provided clinical data for the approval of Pombiliti™, including both experienced and naïve patients.

What is the role of WuXi Biologics in the commercialization of Pombiliti?

WuXi Biologics serves as the exclusive commercial drug substance manufacturing partner for Pombiliti™, utilizing its global sourcing strategy.