Amicus Therapeutics Quest To Cure Rare And Orphan Diseases

Amicus Therapeutics is a company in the biotechnology industry that is headquartered in Cranbury, New Jersey. It is a publicly traded company (Nasdaq: FOLD) that had its initial public offering in 2007. Prior to going public early investors in Amicus Therapeutics were New Enterprise Associates, Radius Ventures, and Canaan Partners.

It is providing cures for rare and orphan diseases that Amicus Therapeutics has as its mission. One set of disorders they are particularly focused on providing a cure for are lysosomal storage disorders. The platform that this company uses to develop products is Chaperone-Advanced Replacement Therapy (Seeking Alpha). They have a number of enzyme replacement therapies in various stages of development.

At Amicus Therapeutics they have three medical issues in particular that they are working on. These are Fabry disease, Epidermolysis Bullosa, and Pompe Disease. They have five drugs under development. The ones furthest along are for Fabry Disease. They have one drug, Galafold (Migalastat) Monotherapy that has been approved for use in the European Union. SD-101 is a topical drug being developed for Epidermolysis Bullosa that is in Stage 3 of development. For Pompe Disease the drug under development is ATB200/AT2221 which is in Phase 2.

Over the years Amicus Therapeutics has received grants in support of the diseases it is seeking to cure. Two of these occurred in 2010. the Michael J. Fox Foundation gave a $500,000 grant in that year while the Alzheimer’s Drug Discovery Foundation granted $210,300.

Amicus Therapeutics is led by Chairman of the Board and Chief Executive Officer John Crowley. He has been in the biotechnology field for almost 20 years. Another top leader of the firm is Bradley Campbell who is the President and Chief Operating Officer. Among the other senior executives are Chief Financial Officer Chip Baird and two Chief Science Officers, Hung Do Ph.D. and Jay Barth M.D.