The Fight against Rare and Orphan Diseases at Amicus Therapeutics

As the world evolves, there emerges different kind of diseases with different treatment requirements. These diseases can at some point prove to be stubborn thus needing special attention by the health organization. There is much need of active commercial organizations and sufficient clinical programs to initiate every possible means of fighting these of diseases. Currently, there are plenty of patients trapped in the rare disease community who need intensive care but are suffering because of either lack of knowledge or lack of enough care facilities. However, there are few available therapeutic centers such as the Amicus Therapeutics that offer services on such rare diseases.


Amicus Therapeutics attained its publicity under the NASDAQ trading symbol FOLD (YahooFinance). The biopharmaceutical company is an American public facility based in Cranbury New Jersey. Amicus Therapeutics was founded in the year 2002 by John Crowley and is focused mainly on rare and orphan diseases, collectively known as lysosomal storage disorders. The company previously received financial supports from various venture capital firms such as; radius ventures, Canaan Partners, and new enterprise associates among others. To achieve higher goals in its therapeutic fields, Amicus Therapeutics did a three years deal collaboration with GlaxoSmithKline and JCR Pharmaceutical. The collaboration was as a result of the advancement of Migalastat Monotherapy  and was to investigate co-formulation with recombinant alpha galactosidase


According to sources, Amicus Therapeutics is a contract manufacturer oriented company, it does not manufacture on its own drugs. The company has to receive support from other prominent organizations such as the Shire Company. Despite its financial mayhems, the company managed to put up another research site branch in San Diego in the year 2008. At some point, the company faced financial difficulties; this was due to the termination of the long term financial relationship with Shire Company in the year 2001. The company nearly lost direction since the Shire agreement termination led to a massive loss of shares.


With the collaboration of David Geffen School of medicine, Michael J. Fox Foundation donated U.S$ 500,000 for study conduct support. In addition, U.S 210,300 was granted to the firm with Alzheimer Drug Discovery Center in collaborations with Icahn School of Medicine ( After that, Amicus Therapeutic managed to acquire various medical centers such as the Calliddus Biopharma in 2013 and Sciodem in 2015 that cost about U.S $947 million both in cash and stock.

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