Amicus Therapeutics – Focusing on Rare and Orphan Illness

Amicus Therapeutics is a biopharmaceutical company that is based in Cranbury in New Jersey. It’s an American public company which was founded in 2002, and it became public in 2007 under NASDAQ trading symbol. Before their IPO, Amicus Therapeutics was funded by several venture capital firms such as Canaan Partners, New Enterprise Associates and Radius Ventures.


Amicus Therapeutics focuses mainly on rare and orphan illness. They specialize mainly in disorders largely known as lysosomal storage disorders. The development of the company’s product is largely dependent on the CHART. In 2014, Amicus Therapeutics gained the recognition of being having with no doubt the largest small molecule pharmacological chaperones portfolio within the pharmaceutical industry.


By Feb. 2014, Amicus Therapeutics had no retailed products. It most marketed product was migalastat whose trade name is Galafold (AliveNewspaper). Migalastat is a pharmacological chaperone that is used in the treatment of Fabry disease. The main aim of Migalastat is to stabilize the endogenous mutant alpha-galactosidase. To advance migalastat monotherapy, Amicus Therapeutics decided to partner with the giants in the pharmaceutical world such as GlaxoSmithKline and JCR Pharmaceutical to look into the matter of coformulation with recombined alpha-galactosidase.


The association between Amicus Therapeutics and GlaxoSmithKline and JCR Pharmaceutical was in existence for about three years that is from 2010 to 2013. Since Amicus Therapeutics had no manufacturing ability of its own, it was forced to rely only on contract manufacturing. In 2008, the company had good fortune, and it was able to expand its operations from New Jersey and opened another site for operation in San Diego.


Unfortunately, the following year the company suffered a great financial setback. That resulted from the fact that the multi-year partnership agreement they had made with The Shire was terminated. During the termination time, financing of the operations was mainly done through private placements and from the previous partnership with The Shire.

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