The Dr. Sol J. Barer Award for
Vision, Innovation and Leadership
Loading the player ...
RareVoice Lifetime Achievement Award –
Rare Disease Congressional Caucus Briefing
Home >> About Us >> Overview
Amicus Therapeutics (Nasdaq:FOLD) is a biotechnology company at the forefront
of advanced therapies to treat a range of devastating 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.
Amicus is seeking global approvals of its lead product candidate, migalastat, a
personalized medicine in late-stage development to treat individuals with Fabry disease on the basis of their genetic diagnosis. Zorblisa, a product candidate in late-stage development, is a potential first-to-market therapy for the chronic, rare connective tissue disorder Epidermolysis Bullosa (EB).
The Company is leveraging its biologics and
Chaperone-Advanced Replacement Therapy (CHART™)
platform technologies to develop next-generation ERT products
for Fabry, Pompe and other lysosomal storage disorders (LSDs).
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 a next-generation Fabry ERT.
Amicus Therapeutics is committed to improving the lives of patients and families affected by rare and orphan diseases.