How Amicus Therapeutics Seeks To Help Those With Rare And Orphan Diseases

Amicus Therapeutics, Inc. is a biotechnology firm that is headquartered in Cranbury, New Jersey. The goal of this company is to find cures for disease that are rare and orphan in nature. The main diseases they are currently working to provide cures for are Fabry disease and Pompe disease, both of which are Lysosomal Storage Disorders. They are also working on drug therapies for Epidermolysis Bullosa which is a connective skin disorder which is genetically acquired.

Amicus Therapies was founded in February of 2002. It is now a publically traded company, having attained its initial public offering in 2007. Its stock trades on the NASDAQ under the symbol FOLD. Before going public it was funded by a number of different venture capital companies. The largest ones backing Amicus Therapies were Canaan Partners, New Enterprise Associates, and Radius Ventures.

John Crowley is the chief executive officer and chairman of Amicus Therapeutics. The other two key executives at the company are Chief Operating Officer Bradley Campbell and Chief Financial Officer William Baird (HighTechChronicle). In addition to its corporate headquarters in Cranbury, New Jersey, Amicus Therapeutics now has offices in other countries around the world. Their international headquarters is located in Buckinghamshire, United Kingdom. In Europe, they have offices in Italy, Germany, France, Spain, and the Netherlands. They also have a presence in Asia as they have offices in Tokyo, Japan.

As Amicus Therapeutics does not have any drug manufacturing facilities it has formed partnerships with other companies in the healthcare industry in order to produce the drugs they make. One of Amicus Therapeutics drugs, Gatefold, has been approved for use in Europe and is on the market. This is a type of Migalastat and treats Fabry disease. Another Migalastat, called Migalastat Monotherapy while in development, is approaching the regulatory phase for release in the United States (Facebook).

Another drug that is in phase 2 trials at Amicus Therapeutics is one for Pompe Disease. This drug is presently called ATB200/AT2221 and so far has shown a lot of promise in treating Pompe disease. Three other drugs are also in the pipeline including one that provides an enzyme replacement therapy.

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