Amicus Therapeutics: Creating Groundbreaking Medical Treatments For Rare Diseases

Amicus Therapeutics is a Cranberry, New Jersey based American biopharmaceutical company. The company focuses on creating treatments for rare, orphan diseases like lysosmal storage disorders. Using a Chaperone-Advanced Replacement Therapy platform, Amicus Therapeutics develops effective enzyme replacement therapies. The company is known for having the pharmaceutical industry’s broadest portfolio went it comes to small molecule pharmacological chaperones. Although Amicus has no marketed products, the pharmacological chaperone treatment migalastat, used to treat Fabry disease, is at an advanced state of development.

Amicus Therapeutics has collaborated with companies like JCR Pharmaceutical, GlaxoSmithKline and Shire to do groundbreaking research and development on effective medical treatments. The company has been trading on NASDAQ under the symbol FOLD since 2007. Prior to that it received funding from New Enterprise Associates, Radius Ventures, Canaan Partners as well as other venture capital firms. Amicus Therapeutics expanded into a second research site in San Diego in 2008. The company doesn’t have its own manufacturing capability. Instead, it relies on contract manufacturing.

The Michael J. Fox Foundation gave Amicus Therapeutics a $500,000 grant in 2010 to help support the company’s collaborative studies with UCLA’s David Geffen School of Medicine. The Alzheimer’s Drug Discovery Foundation gave Amicus Therapeutics a $210,300 grant that same year in support of the pre-clinical work the company was doing at the Alzheimer’s Disease Research Center as part of a collaboration at Mount Sinai with the Icahn School of Medicine.

Amicus Therapeutics acquired Callidus Biopharma, one of it’s competitors in 2013. This gave Amicus the intellectual property and proprietary materials necessary for the creation of the enzyme replacement therapy treatment used for Pompe disease. The company continued to grow in 2015 when it paid cash and stock of $947 million to acquire Scioderm (Google Finance).

One key member of the company’s corporate governance team is John F. Crowley. He has been CEO since 2005 and became chairman of the board in 2010. The COO is Bradley L Campbell and William D. Baird III is CFO. Former board chairman Donald Hayden, Jr. is now the company’s Lead Independent Director. The Chief Scientific Officer of Amicus Therapeutics is David Lockhart.

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