How Amicus Therapeutics Is Researching Helping Those With Rare Diseases

Amicus Therapeutics is a biopharmaceutical company that focuses on rare and orphan diseases. They are headquartered in Cranbury, New Jersey and are led by Chief Executive Officer John Crowley, Chief Operating Officer Bradley Campbell, and Chief Financial Officer William Baird.

One of the disorders that is a particular focus of Amicus Therapeutics is a group of them called lysosomal storage disorders. They are developing enzyme replacement therapies to treat these disorders as well as other diseases. As of 2014 they were notable in the pharmaceutical field as having perhaps the most comprehensive small molecule pharmacological chaperones portfolio in the entire industry.

As Amicus Therapeutics does not have the ability to manufacture drugs themselves they have so far relied on contract manufacturing in order to develop their drug candidates. They did expand, though, in 2008 and now have a second research site, in addition to their one in Cranbury, which is located in San Diego, California.

Amicus Therapeutics work has been supported by a number of national non-profits. The Michael J. Fox Foundation granted the company $500,000 in 2010 so that they could collaborate with the David Geffen School of Medicine at UCLA on a joint project. In that same year, they also received $210,300 from the Alzheimer’s Drug Discovery Foundation for their work. This work involves a joint project with the Icahn School of Medicine at Mount Sinai and is searching for a cure for Alzheimer’s.

In addition to lysosomal storage disorders, two other diseases are a focus of Amicus Therapeutics ( These are Fabry Disease and Pompe disease. They also research a cure for Epidermolysis Bullosa which is a genetic disorder which affects the connective skin. This research is carried out by Amicus Therapies directly and also through their affiliated company, Scioderm, Inc.

The leading drug that Amicus Therapeutics is creating is called migalastat. This drug is designed to treat those who have Fabry disease . This is their first product to reach late-stage development and if successful will be the first drug on the market to treat this devastating disease.