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 development pipeline
of novel, first-in-class treatments for a broad range of human genetic diseases, with a focus on
delivering new therapies to individuals with lysosomal storage disorders (LSDs).
Amicus is building a leading fully-integrated company in the area of rare diseases. The
Company’s lead product candidate, migalastat, is a small molecule in development for Fabry
disease, both as a monotherapy and in combination with enzyme replacement therapy (ERT).
The Company is also leveraging its biologics and
Chaperone-Advanced Replacement Therapy (CHART™)
platform technologies to develop next-generation ERT products. Its lead biologics
program is ATB200, a uniquely-engineered, next-generation Pompe ERT.
Amicus is applying its
CHART platform to combine ATB200 with a pharmacological chaperone to stabilize
the administered enzyme in blood and potentially improve ERT tolerability and efficacy. Utilizing its
biologics and CHART technologies, Amicus is also developing next-generation treatments for Fabry
and MPS 1 with the potential to expand its pipeline into other LSDs.
Amicus Therapeutics is committed to improving the lives of patients and families affected by rare and orphan diseases.