Amicus Therapeutics Announces Recipients of Inaugural Funding Programme for the Pompe Disease Community

MARLOW, U.K.--(BUSINESS WIRE)--Amicus Therapeutics UK Limited, a subsidiary of Amicus Therapeutics Inc (Nasdaq: FOLD), a patient-dedicated biotechnology company, today announced the first recipients of Amicus Ignite, a community funding programme to support projects that aim to improve the lives of people living with Pompe disease. Amicus Ignite invited applicants from healthcare institutions, patient organisations, academic institutions and charitable organisations, to propose projects addressing areas of greatest unmet need in Pompe. Amicus Ignite applications were open to organisations and projects located outside of the United States.

Nine recipients across Europe and Canada were selected for funding. The recipients included:*

• Spierziekten Vlaanderen, Belgium, for their initiative “Patient (Organisation) Collaboration Day”
• Associazione Italiana Glicogenosi APS, Italy, for their initiative “FlexAbility”
• Institute of Inherited Metabolic Diseases, Paracelsus Medical University Salzburg, Austria, for their initiative “PE² - Prepared and Empowered for Pompe Emergencies”
• Fondazione per la ricerca per le Malattie Neuromuscolari "Un Passo per Te", Italy, for their initiative “CARE-MAP-PD: Mapping Patient Journey to Appropriate Healthcare Facilities in Pompe Disease”
• SphinCS GmbH, Germany, for their initiative “The Stand-Up Project”
• Santiago de Compostela Health Research Institute, Spain, for their initiative “The Importance of Support Groups in Pompe Disease: A Multidisciplinary Digital Platform Powered by AI”
• Fundación de Neurociencias, Spain, for their initiative “Pompe Journey Navigator – A Digital Companion for Patients & Caregivers”
• Guido Baccelli Unit of Internal Medicine, Department of Precision and Regenerative Medicine and Ionian Area, University of Bari Aldo Moro, Italy, for their initiative “A Road Map to Transition to Adult Service for Pompe Disease Patients”
• Canadian Association of Pompe, Canada, for their initiative “Pompe Starts with PE: Recognising Early Signs in Children”

“Funding initiatives like Amicus Ignite can play a vital role in driving meaningful improvements for people living with rare diseases such as Pompe disease. The calibre and creativity of the submissions were truly outstanding, showcasing the ambition, diversity, and depth of thought dedicated to addressing unmet needs within the Pompe community. I’m excited about the progress being made and the positive impact these initiatives will have on people affected by Pompe,” said Professor Peter (Pitt) Young, Clinical Director of Neurology at Medical Park, Bad Feilnbach Reithofpark, Germany and Amicus Ignite Judging Panel Member.

A panel of experts, including clinicians, patient organisation representatives, and Amicus stakeholders, carefully reviewed applications against key evaluation criteria. Applications related to treatment or medicinal products were prohibited.

“Pompe disease is a rare genetic disease which leads to progressive muscle weakness, respiratory and heart issues. It can have a significant impact on quality of life and cause multifaceted challenges for patients and caregivers. Amicus Ignite supports initiatives that are innovative, scalable, replicable and sustainable, with the potential to make a tangible difference to the lives of people living with Pompe,” said Tiffany Patrick, Chief Patient Officer of Amicus Therapeutics, Inc.

For more information about the Amicus Ignite programme and the funded projects, visit ignite.amicusrx.co.uk.

*Funded and contracted recipients are included in this announcement.

About Pompe Disease

Pompe disease is an inherited lysosomal disorder caused by a 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 on 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 in skeletal muscles and muscles controlling breathing.

About Amicus Therapeutics

Amicus Therapeutics UK Limited, a subsidiary of Amicus Therapeutics Inc. (Nasdaq: FOLD), a global, patient-dedicated biotechnology company focused on discovering, developing and delivering medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of medicines for rare diseases. For more information, please visit the company’s website at www.amicusrx.co.uk