Amicus Therapeutics – Developing Drugs for Rare and Orphan Diseases

The world of pharmaceuticals and biotechnology is fast growing, and many companies are trying to makes its way to the top. Thanks to the efforts undertaken by these companies, cure, and treatment for many of the diseases are now available, which positively helps millions of people across the globe. One of the biopharmaceutical companies in the United States that have been making rapid progress in the last few years is Amicus Therapeutics. It is a publicly traded firm that trades under the symbol of FOLD. Previously, the company was to trade under the symbol AMTX, but the enterprise’s management later withdrew that decision. The company was funded by many of the large scale venture capital firms, such as Radius Ventures, Canaan Partners, and New Enterprise Associates.


Amicus Therapeutics is known well in the field of finding a cure and developing drugs and therapies for Lysosomal storage disorders and rare and orphan diseases. The company has made considerable progress in the last few years. Some of the drugs developed by Amicus Therapeutics are already licensed and approved, while others are soon going to be approved and waiting for approval. The drug development mechanism used by Amicus Therapeutics is highly advanced and considered to be the safest in the medical world. Amicus Therapeutics follows the mechanism known as Chaperone Advanced Replacement Therapy. The company at the moment does not have its manufacturing unit and relies completely on contract manufacturing to produce drugs. Amicus Therapeutics is also known for developing medicines and therapies for the specific disorders related to enzyme replacement.


Amicus Therapeutics started its operations when the company received grants of $500,000 from Michael J Fox Foundation and $210,000 from Alzheimer’s Drug Discovery Foundation ( The company also received a considerably sized grant from the David Geffen School of Medicine. The primary focus of Amicus Therapeutics was to find an effective cure for the rare and orphan diseases that are known to take lives of millions of people and infants every year. The primary product marketed by the company at the moment is Migalastat, which is known to help cure the rare disease named Fabry Disease.

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