Amicus Therapeutics is an international biotechnology company that offers therapies for the orphan and rare diseases. It is a public biopharmaceutical based in Cranbury, New Jersey. Amicus Therapeutics became a public corporation in 2007 under the NASDAQ trading symbol FOLD. Before going public, the company received funding from New Enterprise Associates, Canaan Partners, and Radius Ventures. The biotech’s therapeutic focus is on the lysosomal storage disorders which are the rare and orphan diseases (,-NJ-jobs.html). Other than its single site located in New Jersey, the company also has another research site in San Diego. The CEO of the corporation is John Crowley who has served in this position since 2005.


In 2010, Amicus Therapeutics partnered with JCR Pharmaceutical to carry out a study on co-formulation with recombinant alpha-galactosidase. Currently, the company is leveraging its biologics as well as CHART to advance the ERT products for Pompe disease, Lysosomal Storage Disorders and Fabry disease. Amicus Therapeutics’ top biologics program is the ATDB200/AT2221 which is a Pompe disease ERT that is set to be administered together with the pharmacological chaperone. The product development cycle of Amicus is based mainly on CHART i.e. Chaperone-Advanced Replacement Therapy. It also focuses more on the advancement of enzyme replacement therapies, ERTs.


By February 2014, Amicus had not launched any marketed products. Up to date, Amicus Therapeutics does not manufacture its products but rather relies on outsourced contracts. Its lead product candidate is migalastat, also known as Galafold. It is a pharmacological chaperone treatment which works as a personalized medicine in late stage development to treat Fabry illness based on genetics diagnosis. This medicine works by stabilizing endogenous mutant alpha-galactosidase. Three years ago, Amicus Therapeutics was recognized for its broad portfolio of small molecule pharmacological chaperones.


Before Crowley took up the role of CEO in Amicus, the company was led by Donald Hayden as the Chairman (Twitter). He continued to serve the company as the Lead Independent Director as Crowley took up the CEO role. The chief scientific officer of the Amicus Therapeutics is David Lockhart, and the chief financial officer is William D. Baird. The executive team also comprises of Brandon Wustman who is the Director of Exploratory Biology and COO, Bradley L. Campell.