Pombiliti + Opfolda (AT-GAA)
Pompe Disease (IOPD, 0 to <18 yrs)
Key Facts
About Amicus Therapeutics
Amicus Therapeutics is a fully integrated, global biotech with a mission to address the vast unmet need in rare diseases, where 95% of over 10,000 known conditions lack approved treatments. The company has achieved commercial success with two approved therapies for Fabry and Pompe disease, validating its dual-technology platform. Its strategy centers on deepening its impact in core lysosomal disorders, expanding into adjacent rare diseases like FSGS, and maintaining a patient-centric culture that drives innovation and operational execution.
View full company profileAbout Amicus Therapeutics
Amicus Therapeutics is a fully integrated, global biotech with a mission to address the vast unmet need in rare diseases, where 95% of over 10,000 known conditions lack approved treatments. The company has achieved commercial success with two approved therapies for Fabry and Pompe disease, validating its dual-technology platform. Its strategy centers on deepening its impact in core lysosomal disorders, expanding into adjacent rare diseases like FSGS, and maintaining a patient-centric culture that drives innovation and operational execution.
View full company profile