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Amicus Therapeutics: A Leader in the Treatment of Rare Diseases

Amicus Therapeutics: A Leader in the Treatment of Rare Diseases

Amicus Therapeutics has its headquarters office in Cranbury, New Jersey. The international headquarters office of Amicus is located in the United Kingdom. The other locations of Amicus Therapeutics are located in France, Germany, Italy, Netherlands, and Spain. It became a publicly traded company in 2007. The stock symbol of Amicus is FOLD. Amicus is traded on the NASDAQ market. The company was established in February 4, 2002. John F. Crowley has been the CEO of Amicus Therapeutics since 2005. Bradley Campbell is the Chief Operating Officer. William “Chip” Baird is the Chief Financial Officer. As of December 31, 2013, there were 92 employees at Amicus Therapeutics (YahooFinance).


The therapies offered by this pharmaceutical company focus on the care of rare and orphan diseases, such as lysosomal storage disorders (LSDs). SD-101 was developed for the treatment of Epidermolysis Bullosa (EB), a rare tissue disease that causes skin blistering. Migalastat was created for treating Fabry disease. It stabilizes the alpha-Gal A produced in the patient’s cells so it can be moved to the lysosomes. When it gets to the lysosomes, the GL-3 could be reduced. The brand name for Migalastat is Galafold. In order to develop enzyme replacement technologies(ERTs), Amicus Therapeutics leverages the Chaperone-Advanced Replacement Therapy (CHART) technology. Also, CHARTs are used to make ATB200/AT222, a treatment method consisting of a recombinant human acid alpha-glucosidase (rhGAA) enzyme. Therefore, Pompe disease can be treated with ATB200/AT222 because the deficiency of GAA is eliminated with rhGAA enzymes. As of right now, Amicus Therapeutics is discovering a new protein replacement therapy for CDKL5 (cyclin-dependent kinase-like 5) deficiency.


The company uses contractors for manufacturing because it is not an independent manufacturer. Amicus Therapeutics bought Callidus Biopharma in November 2013 (https://forexwizard.biz/2017/09/15/amicus-therapeutics/). As a result of the acquisition, Amicus had access to proprietary items and intellectual property that would be useful for the enzyme replacement technology used in treating Pompe Disease. The company made another acquisition in September 2015. This time it bought Scioderm for $947 million. CabaRare is a festival hosted by Amicus to celebrate RareDisease day. Performers with rare diseases participate in this festival.

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