Amicus Therapeutics is a public American biopharmaceutical company based in Cranbury, New Jersey.:2 The company went public in 2007 under the NASDAQ trading symbol FOLD. This followed a 2006 planned offering and subsequent withdrawal, which would have established the trading symbol as AMTX Prior to their IPO, Amicus was funded by a variety of venture capital firms including Radius Ventures, Canaan Partners and New Enterprise Associates. The therapeutic focus of Amicus is on rare and orphan diseases, particularly disorders collectively called lysosomal storage disorders.:2 The companys product development is based largely on the Chaperone-Advanced Replacement Therapy (CHART®):18 platform and has concentrated on development of enzyme replacement therapies (ERTs).:2 In 2014, Amicus was noted as having, arguably, "the broadest portfolio of small molecule pharmacological chaperones" in the pharmaceutical industry.:95 As of February 2014[update], the company had no marketed products, its most advanced candidate being migalastat (trade name Galafold), a pharmacological chaperone treatment for Fabry disease which aims to stabilize endogenous mutant alpha-galactosidase.:5 In parallel with advancement of migalastat monotherapy, the company engaged in a collaboration with GlaxoSmithKline and JCR Pharmaceutical to investigate coformulation with recombinant alpha-galactosidase (JR-051).:7 This collaboration lasted for three years, from 2010 to 2013.
Highest paying job titles at Amicus Therapeutics include Director of Regulatory Affairs, Director of Biostatistics , and Patient Safety Director