Bradley Campbell, President and Chief Operating Officer of Amicus Therapeutics, Inc., stated, “This approval of Galafold is a transformative moment for the Fabry community in the EU, as it gives those patients as young as 12 years of age with an amenable mutation a new treatment option for the first time in more than 15 years. We are grateful to this wonderful and passionate community, particularly the patients, physicians, and families who have made this research possible through their participation in the clinical trials, as well as to the regulators and our dedicated Amicus team. This achievement highlights our company’s mission, which is focused on transforming the lives of people living with rare diseases.”
“This expanded approval is a significant step forward for the Fabry community, as we work towards increasing awareness of this rare disease in young people. It ensures patients, both paediatric and adult now have a convenient oral disease modifying therapy option available,” said Uma Ramaswami, MD, Lysosomal Storage Disease Unit, Royal Free London NHS Foundation Trust.
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