Amicus Therapeutics Making a Difference in the Lives of Patients

Amicus Therapeutics is a biotechnological company recognized globally for their dedication towards the treatment of orphan diseases. The company has been at the forefront of research and treatment of a range of devastating rare diseases.

Amicus Therapeutics was founded in February 2002 and is based in Cranbury, New Jersey. The company is headed by John Crowley, who is the CEO and Chairman of the board. Other key figures include Bradley Campbell, who is the Chief Operations Officer, and William Baird, who is the Chief Finance Officer. Amicus Therapeutics is a publicly traded company under the brand NASDAQ. The company has been on the stock exchange since 2007 after a planned IPO.

Amicus mainly focuses on rare and orphan diseases (http://www.phillypurge.com/2017/08/02/amicus-therapeutics-the-fight-against-the-rare-diseases/). The Chaperone-Advanced Replacement Therapy, otherwise referred to as CHART is the main basis of the company’s product development. Amicus has put an emphasis on enzyme replacement therapies (ERTs). The company’s efforts have earned them accolades. In 2014, Amicus was recognized as the company with the broadest portfolio in terms of small molecule pharmacological chaperones in the entire pharmaceutical industry.

Although they had no marketed products by February 2014, some of the most notable developments from Amicus Therapeutics include migalastat, otherwise known as Galafold. Galafold is a pharmacological chaperone treatment which is aimed at stabilizing endogenous mutant alpha-galactosidase for the treatment of Fabry disease.

Amicus Therapeutics has no manufacturing capabilities and therefore relies on contract manufacturing (WeeklyOpinion). Additionally, the company collaborates with other like-minded firms to come up with treatments for complex diseases. One of its most notable collaborations was with GlaxoSmithKline and JCR Pharmaceutical. The collaboration which lasted from 2010 to 2013 was aimed at investigating co-formulation with recombinant alpha-galactosidase.

It is worth noting that in 2008, the company expanded its activities from its single location in New Jersey to a second research facility in San Diego. Additionally, Amicus got a big financial boost, when they received a US$500,000 grant in 2010 from the Michael J. Fox Foundation. The grant was aimed at supporting studies conducted by Amicus in collaboration with the David Geffen School of Medicine.

In 2013, Amicus went a step further and acquired CallidusBiopharma, which was one of their competitors. The acquisition granted Amicus Therapeutics intellectual and material property for the treatment of Pompe disease.

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