How Amicus Therapeutics Is Helping Those With Rare And Orphan Diseases

Amicus Therapeutics is headquartered in Cranbury, New Jersey. It is a biopharmaceutical company which is publicly traded under the stock ticker FOLD. It went public in 2007 and prior to that had received funding from three venture capital companies, New Enterprise Associates, Canaan Partners, and Radius Ventures.

The type of cures that Amicus Therapeutics specializes in is devastating rare and orphan diseases ( They are developing enzyme replacement therapies which have shown a great deal of promise in helping patients with these types of diseases.

In order to help Amicus Therapeutics develop their cures, the Michael J. Fox Foundation had granted them $500,000 in 2010 (YahooFinance). This money was provided in a partnership with the David Geffen School of Medicine at UCLA. That same year they also received money from other sources such as the Alzheimer’s Drug Discovery Foundation who granted $210,300. In further support of their important research Amicus Therapeutics acquired Callidus Biopharma in 2013 which gave them access to both intellectual property as well as proprietary materials.

John F. Crowley is the Chief Executive Officer of Amicus Therapeutics, a position he has held since 2005. He is also the company’s Chairman of the Board. Other top executives at this company include Bradley L Campbell, who is the Chief Operating Officer, and William D. Baird III who is the Chief Financial Officer. Amicus Therapeutic’s critical Chief Scientific Officer position is held by David Lockhart.

Three of the diseases that Amicus Therapeutics is developing treatments for is Fabry Disease, Epidermolysis Bullusoa, and Pompe Disease. They have seven drugs in various stages of development. One is Galafold Monotherapy which treats Fabry Disease. This drug has been approved in the European Union and is awaiting FDA approval in the United States. Their other drug that is closest to approval is another one which treats Fabry Disease called Migalastat Monotherapy which is in Phase 3 development.

Epidermolysis Bullosa is a genetic condition which is a connective skin disorder. Amicus Therapeutics has a drug just entering Phase 3 development called SD-101 which will be a topical formulation that patients spread on their skin in the affected areas.


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