Amicus Therapeutics is an international biotechnology company that that was established in 2002. The firm became publicly traded in 2007, and it has been using FOLD as its trading symbol. FOLD is listed on NASDAQ. It has specialized in the development of cures for various rare diseases. The disorders that it deals with include Pompe and Fabry (Epidermolysis Bullosa), which is a genetic connective skin condition. The head offices of the corporation are in Cranbury, New Jersey.


The biotechnology firm has to date developed several products. Its lead candidate product called migalastat, and it is currently in its last stage of development. The company has also created another therapy that is known as SD-101 and is focused on treating Epidermolysis Bullosa. Amicus Therapeutics also has a lead biologics plan that is known as ATB200/AT2221 and has been developed to cure the Pompe disease. The drug is administered jointly with a pharmacological chaperone to the patient. The company has used Chaperone-Advanced Replacement Therapy in developing ATB200/AT2221. It is also determined to create excellent enzyme replacement therapies.


Amicus Therapeutics has been appreciated for leading in the development of the therapies for curing rare genetic disorders. The firm has been giving hope to patients who suffer from extraordinary diseases. Its drugs have been approved by EMA, and they are awaiting FDA licensing this year. In 2017, the company has three main goals, and they are the forwarding J-NDA for migalastat, creating an alternative clinical ATB200/AT2221 to cure Pombe disease, and conducting a phase 3 clinical trial for Epidermolysis Bullosa.


The ATB200/AT221 plan uses an exceptional treatment mechanism. ATB200 is a useful recombinant alpha-glucosidase enzyme, and it contains mannose-6 phosphate moieties that facilitate its uptake. AT221 is a chaperone that is combined with ATB200 to create a compound that is stable. According to Amicus Therapeutics, the program will offer it a 12 percent share in the world’s rare disease treatment field (YahooFinance). It has estimated to gain a $1.2 billion market from the Pombe program. The company is served by a team of scientists who are devoted to offering the best treatments to patients across the globe. It is committed to targeting mutated proteins when developing its therapies.

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