Posts in Category: Medical

The Medicine and Technology of Amicus Therapeutics

Amicus Therapeutics is a company based in Cranbury, New Jersey. It specializes in treating a variety of rare and orphan diseases. The diseases this company focuses the most on are called lysosomal storage diseases, which are a metabolic disorder that comes from defects in the function of the lysosomal. This company has concentrated on creating ERT’s (enzyme replacement therapies). In 2008 the company made a choice to expand its research capability by adding a second site in San Diego, California.


The mission of Amicus Therapeutics is to drive towards healing and innovation. They are committed to bringing impactful benefits to the community of rare and orphan diseases, as well. There are three main beliefs of Amicus Therapeutics: fight to stay at the forefront of therapies for orphaned diseases, create long term values for their stakeholders, and believe in each other in order to build respect and teamwork for every employee’s contribution.


The lead product candidate Amicus Therapeutics has is called migalastat. It is a personalized development to cure individuals with Fabry disease based on their genetic diagnosis. Another example of a product candidate is called SD-101. This is a product that is new to the market therapy which treats (EB) or Epidermolysis Bullosa. It is a rare, genetic connective tissue disorder ( The companies lead biologics program is called ATB200/AT2221. This is a Pompe disease ERT which becomes combined with a pharmacological chaperone. ERT stands for Estrogen replacement therapy. This is a form of therapy where hormones treat the symptoms of menopause. One example of the symptoms is disruption of sleep.


There was a recent communication written by the FDA (“Food and Drug Administration”) to Amicus Therapeutics (YahooFinance). They told Amicus that they were allowed to submit an NDA (“New Drug Application”) for a migalastat. This will take effect at the conclusion of 2017.