Amicus Therapeutics and the Treatment of Rare Diseases

Amicus Therapeutics is a biotechnology company that focuses on researching treatment for rare and orphan diseases. Although the company is based in Cranbury, New Jersey, they operate a second research site in San Diego, California. Amicus was founded in 2002 but didn’t go public until 2007. The company is fairly small, with around five hundred employees. Amicus is primarily research based and has to have other companies take care of manufacturing. Although the company offers no products as of 2017, a few potential products are in the final stages of being approved for commercial use.

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Fabry Disease is one illness that Amicus Therapeutics is currently researching. The company has developed two potential products to help treat it: Galafold/Migalastat Monotherapy and novel ERTs (Enzyme Replacement Therapy). Galafold is an orally administered monotherapy that has full approval in Europe and is ready to be released to the public. Migalastat Monotherapy and Galafold are one in the same, with Galafold being the brand name. While the product is ready for use in Europe, it is still in the approval stages in the Americas and the rest of the world. Amicus’ ERTs for Fabry Disease are in preclinical stages of research.
In addition to Fabry Disease, Amicus Therapeutics studies two other illnesses specifically, Pompe Disease and Epidermolysis Bullosa. Their product for Pompe Disease, a novel ERT named ATB200 and AT2221 are in the middle of the final phase of testing. This treatment will require a chaperone as well. Amicus’ medication for Epidermolysis Bullosa is named SD-101. It is a topical medication. Currently, SD-101 is in the third and final phase of testing.
While Amicus Therapeutics focuses on a few diseases specifically, they also conduct research on other rare diseases and plan treatments for patients with these illnesses. Most of their medications for these various diseases are novel ERTs and most of them are in the primary research phase of development.
Dedicated to research and creating treatments for those with rare illnesses, Amicus Therapeutics is a company to keep an eye on. Several products are nearing their final stages and are one step closer to helping others survive.
More here: Crunchbase