Amicus Therapeutics is a leading American biopharmaceutical company. They are headquartered in Canbury, New Jersey and have been listed on the NASDAQ stock exchange, under the symbol “FOLD,” since 2007 (YahooFinance). The company was founded in 2002 and is led by Chairman & CEO John Crowley, a position he has held since 2005.
Amicus Therapeutics focuses its research and development efforts into treating rare and orphan diseases. In much of the world, these “orphan diseases” are so rare that there is no market for a therapy to be developed, meaning that a government agency or non-profit non-governmental agency (NGO) will often fund the research into developing treatments for the diseases.
There is not an ironclad definition of what constitutes a “rare” disease, but since most of them are genetic, the organization Global Genes keeps track of them. Global Genes estimates that over 7,000 diseases affect more than 300 million people worldwide.
Amicus Therapeutics is in the process of developing treatments for the rare genetic disorder Fabry disease. The company’s lead product candidate, migalastat, has been approved for use in the European Union and is far along in the process for the United States and the rest of the world. The company also has other treatments in varies stages of production, including treatments for Epidermolysis Bullosa and Pompe Disease. Amicus Therapeutics also is developing Enzyme Replacement Therapy as well.
In addition to the treatments, Amicus Therapeutics has developed a proprietary technology called CHART. CHART stands for Chaperone-Advanced Replacement Therapy and is used in the company’s efforts in research into enzyme replacement therapies. This breakthrough technology has allowed Amicus Therapeutics to create SD-101, a product candidate that would be first-to-market in the treatment of Epidermolysis Bullosa.