The Dr. Sol J. Barer Award for
Vision, Innovation and Leadership
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Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront
of developing therapies for rare and orphan diseases. The Company has a robust pipeline
of novel, first-in-class, small molecules called pharmacological chaperones for
the treatment of lysosomal storage diseases (LSDs). These chaperones may offer a
dual-treatment approach for Fabry, Pompe, Gaucher and other LSDs. As orally administered monotherapy agents, pharmacological
chaperones are designed to bind to, stabilize and increase the activity of a patient’s own misfolded enzyme.
In combination with enzyme replacement therapy (ERT), pharmacological chaperones
may improve the uptake of the infused enzyme and potentially improve ERT outcomes.
Amicus is developing its lead pharmacological chaperone migalastat HCl for Fabry
collaboration with GlaxoSmithKline (GSK), and owns exclusive global rights
to the rest of its pipeline and technology platform. Phase 3 studies of migalastat
HCl monotherapy (Study
Study 012) and a Phase 2 study of migalastat HCl co-administered with
013) are currently underway. A second Phase 2 study (Study
010) is ongoing to evaluate the pharmacological chaperone AT2220 (duvoglustat
HCl) co-administered with ERT for Pompe disease. Amicus is also conducting preclinical studies of pharmacological chaperones in combination with ERT for Gaucher disease and other LSDs.
In addition to LSDs, pharmacological chaperones may have the potential to treat neurodegenerative disorders
and other human genetic diseases caused by misfolded proteins.
Amicus Therapeutics is committed to improving the lives of patients and families affected by rare and orphan diseases.