The Dr. Sol J. Barer Award for
Vision, Innovation and Leadership
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RareVoice Lifetime Achievement Award –
Rare Disease Congressional Caucus Briefing
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Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront
of therapies for rare and orphan diseases. The Company has a robust pipeline
of novel, first-in-class treatments in development for a broad range of human genetic diseases, with a focus on
delivering new benefits to individuals with lysosomal storage diseases (LSDs).
Amicus’ lead small molecule pharmacological chaperone migalastat HCl
for Fabry disease is being developed in collaboration
with GlaxoSmithKline (GSK). As an oral monotherapy migalastat HCl is in Phase 3 for Fabry patients
who have genetic mutations that are amenable to this chaperone in a cell-based assay. For all other individuals
with Fabry disease, Amicus and GSK are developing migalastat HCl co-formulated with a proprietary Fabry enzyme
replacement therapy (ERT).
Outside the GSK collaboration, Amicus retains worldwide rights to the rest of its pipeline and proprietary
Chaperone-Advanced Replacement Therapy (CHART™) platform. Amicus is leveraging the CHART platform to
develop pharmacological chaperones co-administered with currently marketed ERTs as well as next-generation
therapies that consist of proprietary lysosomal enzymes co-formulated with pharmacological chaperones.
Current CHART programs for Pompe disease include the pharmacological chaperone AT2220 (duvoglustat HCl)
co-administered with ERT in Phase 2, as well as a next-generation therapy in preclinical development.
Additional CHART programs are in preclinical development for Gaucher disease and other LSDs.
Amicus Therapeutics is committed to improving the lives of patients and families affected by rare and orphan diseases.