Amicus Therapeutics: Leveraging the Genetic Revolution

Amicus Therapeutics: Leveraging the Genetic Revolution

Amicus Therapeutics is a pharmaceutical company headquartered in Cranbury, NJ and dedicated to developing treatments for rare and orphan diseases. The company was founded in 2002 and has been publically traded on the NASDAQ since 2007. John F. Crowley has served as the CEO since 2005.


Pharmaceutical investment in rare diseases has always been limited because it is difficult to justify research and development expenses when the potential market for a drug is small. Modern biotechnological advances, however, allow innovative companies like Amicus Therapeutics to develop treatments for these diseases at a fraction of the cost of traditional, brute-force approaches. As such, Amicus Therapeutics currently has a pipeline of several promising pharmaceuticals that are expected to reach the market in the coming years.


Furthest along in development is Migalastat, which is already approved for use in patients in the European Union. Amicus Therapeutics has developed Migalastat as a personalized-medicine treatment for the systematic genetic condition known as Fabry disease. Personalized medicine means that a specific biological pharmaceutical (i.e., a protein) given to each patient is custom-designed based on the genetic abnormalities causing their particular manifestation of the disease.


The product candidate SD-101, which is in phase-three clinical trials, could become the first commercially available treatment for Epidermolysis bullosa, a genetic disease of the connective tissue. This condition, which occurs in only nine out of every million people in the general population, highlights the commitment of Amicus Therapeutics to tackling truly rare diseases (GoogleFinance).


Amicus Therapeutics has, in stage-two clinical trials, an enzyme-replacement therapy, currently referred to as ATB200/AT2221, for Pompe disease. This condition results in the buildup of excess glycogen around the body’s cells and is caused by a deleterious genetic mutation in the enzyme alpha-glucosidase. The therapy for Pompe disease, and for other diseases being investigated by Amicus Therapeutics, involves replacing these defective enzymes with correct versions synthesized in the laboratory.


The primary goal of the Human Genome Project was to obtain insights leading to new frontiers in medicine. With just 92 employees, small and nimble Amicus Therapeutics is now leveraging the tools of the genetic revolution to make this vision a reality.

More about Amicus Therapeutics at