Amicus Therapeutics: Battling Rare Diseases

Amicus Therapeutics is an American pharmaceutical company that is headquartered in Cranbury, New Jersey. Its shares are currently available on the stock market under the trading symbol FOLD, and the company has been gathering additional funding through venture capital firms. It was founded in February 4, 2002, and the company was established to focus on rare diseases that affect a small percentage of the population. One of these rare diseases that Amicus Therapeutics is trying to study is called the lysosomal storage disorder. It is a group of at least 50 inherited metabolic diseases, and its occurrence is very rare. It results to defects in lysosomal function, and there is no available cure. Amicus Therapeutics is doing their best to discover a cure for the said disease.

 

Since their establishment, Amicus Therapeutics has been relying on the Chaperone Advanced Replacement Therapy platform and they have been developing an enzyme replacement therapy to combat the disease. Their hard work has resulted into being recognized as the pharmaceutical company who has the widest range of portfolio for small molecule pharmacological chaperone. Amicus Therapeutics does not have any marketed products as of 2014, but they have a pharmacological chaperone treatment called migalastat, which is used to treat a rare disease called Fabry disease. Migalastat’s trade name is Galafold, and the company is trying to promote this treatment by partnering with other companies who would offer it to the public. Amicus Therapeutics is known to have multiple partnerships with other multinational companies, like GlaxoSmithKline, and the researches that they have conducted resulted into the development of effective medicines that can really cure diseases.

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Amicus Therapeutics does not have the capability to manufacture products of its own, but they have contracts with other third party companies when they wanted something to be manufactured. Since their establishment in 2002, they have been expanding, opening up an office in New Jersey and another one in San Diego (Glassdoor). They have been doing a lot of research that is being funded by private foundations like the Michael J. Fox Foundation, and has acquired a number of small competitors, like Callidus Biopharma and Scioderm.

 

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