Amicus Therapeutics Focusing on Treatments for Rare Disease

Americu Therapeutics is a global biotechnology company founded in 2002 and based in New Jersey that focuses on therapies for rare and orphan diseases. The company has a platform that is unique in medicine development and technologies that include diseases and genetic like Disorders Fabry disease, Lysosomal Storage Disorders, Epidermolysis Bullosa the genetic connective skin disorder, and Pompe disease.

In 2004 the company expanded to open a location in San Diego in 2008 and in 2009 the company entered a collaboration agreement with Shire. In 2010 the company received a donation of $500,000 from the Michael J. Fox Foundation to support and collaborate with UCLA Medical School David Geffen in support of studies conducted. Another grant the company received the same year was from the Alzheimer’s Drug Discovery Foundation for support with the Mount Siani Icahn School of Medicine Alzheimer’s Disease Research Center.

https://seekingalpha.com/article/4073303-amicus-therapeutics-small-biotech-seems-big-mo
The company’s product development has been based on the Chaperone-Advanced Replacement Therapy also referred to as CHART. They have been resolute in the development of enzyme replacement therapies starting in 2014. In 2017 the company submitted a Japanese new drug application for migalastat for the treatment of Fabry disease. The company believes with more than 700 Fabry disease patients in Japan the oral medication could benefit their condition. Then another large accomplishment for the company happened in 2017 with the Rare Pediatric Disease designation from the U.S. FDA for SD-101 that was one of the company’s first treatments to get the FDA Breakthrough Therapy designation and then the Orphan Drug designation. This treatment is used in treating the three major types of Epidermolysis Bullosa (EB). This was the first drug for the disease in Phase 3 and that could be the first FDA approved therapy.

Amicus Therapeutics went public in 2007 and listed on the NASDAQ under the trading name FOLD as a pharmaceutical industry. This is a company that employs up to 500 people (YahooFinance). The top people at Amicus Therapeutics are CEO John Crowley, CFO William Baird, and COO Bradley Campbell.
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