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Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscle cells and glycogen buildup causes muscle damage. Disease severity ranges across a spectrum, with predominant manifestations such as skeletal muscle weakness and progressive respiratory involvement.1 Pombiliti + Opfolda is a unique two-component therapy. Pombiliti is a recombinant human GAA enzyme (rhGAA) naturally expressed with high levels of bis-M6P (Mannose 6-Phosphate), designed for increased uptake into muscle cells. Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood. “Today’s FDA approval of Pombiliti and Opfolda is a testament to the power of science, medicine, and our passionate determination to improve the lives of people living with Pompe disease. This approval embodies our Amicus spirit, passion, and resilience and is a very meaningful step for the Pompe community. I am just so immensely proud of our team, and so very grateful to everyone who has worked to bring this medicine to this approval. Most especially to all of the people living with Pompe around the world,” said John F. Crowley, Executive Chairman of Amicus Therapeutics, Inc. Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “The FDA approval of Pombiliti and Opfolda is a major milestone for Amicus. We are grateful to the Pompe community, particularly the patients, caregivers, families, researchers, and physicians who have contributed to the development process through their commitment to our clinical studies. Today’s approval is also a testament to Team Amicus’ extraordinary dedication to patients and our ability to execute on our vision to bring new therapies to the rare disease community. Our highly experienced team is ready to launch this medicine in the U.S., and we look forward to rapidly bringing this new treatment regimen to all eligible adults living with late-onset Pompe disease who are not improving on their current ERT.” The FDA approval was based on clinical data observed from the Phase 3 pivotal study (PROPEL), the only trial in LOPD to study ERT-experienced participants in a controlled setting. “The Pompe community continues to face unmet need and limited treatment options. This two-component therapy is an important new treatment for those adults living with late-onset Pompe disease and not improving on current therapies. I am encouraged by the evidence generated over many years of clinical research studying this therapy for ERT-experienced patients living with late-onset Pompe disease,” said Tahseen Mozaffar, MD, Director of the Division of Neuromuscular Diseases in the Department of Neurology at the School of Medicine at UC Irvine and Director of the UC Irvine ALS and Neuromuscular Center, as well as an investigator for the PROPEL study. “Today’s FDA approval is an extremely important step and acknowledges the potential of Pombiliti and Opfolda,” said Priya Kishnani, MD, Professor of Pediatrics and Chief of Medical Genetics at Duke University School of Medicine and an investigator for the PROPEL study. “I am grateful that eligible patients with late-onset Pompe disease in the U.S. will now have access to additional treatment options.” “The FDA approval of Pombiliti and Opfolda represents a long-awaited day for people living with late-onset Pompe disease and advocating for additional therapeutic options,” said Tiffany House, President, Acid Maltase Deficiency Association. “Amicus’ long-standing commitment to the Pompe community and rare disease research has led to the development of an important therapy for the Pompe community because patients will now have options.” Amicus Therapeutics will launch Pombiliti + Opfolda immediately in the U.S. The FDA previously granted Breakthrough Therapy designation for Pombiliti + Opfolda. Pombiliti + Opfolda has also been approved for the treatment of adults with LOPD in the European Union and the United Kingdom. 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 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. 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.1 About Pombiliti + Opfolda 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. 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 diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. Further information about the Company can be found at: www.amicusrx.com, and can be followed on Twitter and LinkedIn. The content above comes from the network. if any infringement, please contact us to modify.

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. 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). 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. 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. 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. 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." 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. 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." 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 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. 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. About Pombiliti + Opfolda 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. 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 June 30, 2023, WuXi Biologics is supporting 621 integrated client projects, including 22 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. The content comes from the Internet. If there is any infringement, please contact us for deletion.

Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) Approved for Adults Living with Late-Onset Pompe Disease in Great Britain National Institute for Health and Care Excellence (NICE) Issues Final Positive Recommendation Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced that the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom (U.K.) has granted marketing authorizations for Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) for adults living with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency). The indication for Pombiliti is a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). The indication for Opfolda is an enzyme stabilizer of cipaglucosidase alfa long-term enzyme replacement therapy for adults with LOPD. In addition, the National Institute for Health and Care Excellence (NICE) issued final guidance recommending reimbursement of Pombiliti + Opfolda for use within the National Health Service (NHS) in England and Wales. As stated in the guidance, NICE concluded that the cost-effectiveness estimates for Pombiliti + Opfolda showed a positive net health benefit and recommended Pombiliti + Opfolda for adults with LOPD as first line and later lines of therapy. Prior to Great Britain (GB) approval, Pombiliti + Opfolda was granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP), a Priority Innovative Medicines designation, as well as a positive scientific opinion under the Early Access to Medicines Scheme (EAMS). This accelerated the time to market and enabled healthcare professionals to prescribe the treatment prior to marketing authorization based on clinical factors for patients with a clear unmet need. “The MHRA approvals for Pombiliti and Opfolda are a major step forward for adults in the U.K. living with late-onset Pompe who are seeking new treatments. We are grateful to the global Pompe community who have helped advance this therapy, especially the patients, families, and physicians who participated in our clinical studies,” stated Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. “The speed in which NICE recommended reimbursement of Pombiliti and Opfolda is reflective of the U.K.’s Innovative Licensing and Access Pathway, the data behind Pombiliti and Opfolda, the strong collaboration with the reimbursement authorities, and the Amicus commitment to bring this therapy to those living with Pompe disease as quickly as possible. I am proud of Amicus’ relentless commitment toward ensuring patient access to our innovative therapies, and we are working as quickly as possible to make Pombiliti and Opfolda commercially available.” Late-onset Pompe disease (LOPD) is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues. Disease severity ranges on a spectrum, but predominant manifestations are skeletal muscle weakness and progressive respiratory involvement. Prof. Mark Roberts, Consultant Neurologist at the Greater Manchester Neurosciences Unit at Salford Royal NHS Foundation Trust stated, “From the positive uptake of the Early Access to Medicines Scheme, we have already seen the impact that this treatment is having for patients. Having widespread access to this treatment is an exciting development for the Pompe community, giving HCPs and patients a new option that exhibits a novel mode of action.” The MHRA and NICE decisions were based on clinical data from the Phase 3 pivotal study (PROPEL), the only trial in LOPD to study both ERT-naïve and ERT-experienced participants in a controlled setting. Allan Muir, Chair of the Board of Trustees of Pompe Support Network, stated, “Pompe disease is a rare disease, one that extensively affects all aspects of life for an individual and their family, friends, and colleagues. We are very grateful to companies such as Amicus Therapeutics that seek to make meaningful differences for our small community and we welcome new treatments that bring further choice to patients.” Val Buxton, Chief Executive, Association for Glycogen Storage Diseases, stated, “Being able to gain rapid access to effective new therapies via the Early Access to Medicines Scheme has had a meaningful impact on many people affected by Pompe. This approval has provided a much-needed new therapy option for the Pompe community.” Pombiliti + Opfolda has demonstrated fulfilment of the Great Britain orphan designation criteria and will be added to the Orphan Register held by the MHRA. Both will benefit from 10 years of market exclusivity in respect of similar medicinal products in the approved orphan indication. About Pombiliti® + Opfolda® Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa, 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. In clinical studies, Pombiliti + Opfolda 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 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. 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 controlling breathing. 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 diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow us on Twitter and LinkedIn. 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