Description of Business

3 Months Ended

Mar. 31, 2015

Description of Business

Description of Business

1. Description of Business

Corporate Information, Status of Operations, and Management Plans

Amicus Therapeutics, Inc. (the “Company”) was incorporated on February 4, 2002 in Delaware and is a biopharmaceutical company focused on the discovery, development and commercialization of next-generation medicines for a range of rare and orphan diseases, with a focus on improved therapies for lysosomal storage disorders (“LSDs”). The Company’s lead product candidate is a small molecule that can be used as a monotherapy and in combination with enzyme replacement therapy (“ERT”) for Fabry disorder.  The Company’s development programs also include next-generation ERTs for LSDs, including Fabry disorder, Pompe disorder and Mucopolysaccharoidosis Type I (“MPS I”).  The Company’s activities since inception have consisted principally of raising capital, establishing facilities, and performing research and development.

We have completed two Phase 3 global registration studies of migalastat monotherapy.

During the first quarter of 2015, we met with regulatory authorities in Europe and the U.S. to discuss the approval pathways for migalastat as a monotherapy for Fabry patients who have amenable mutations. In Europe, we plan to submit a marketing authorization application (“MAA”) to the European Medicines Agency (“EMA”) in the second quarter of 2015. In the U.S., we plan to submit a new drug application (“NDA”) for accelerated approval (subpart H) with the U.S. Food and Drug Administration (“FDA”) in the second half of 2015.

In November 2014, the Company issued a total of 15.9 million shares through a public offering at a price of $6.50 per share, with net proceeds of $97.2 million. The Company expects to use the net proceeds of the offering for investment in the global commercialization infrastructure for migalastat monotherapy for Fabry disorder, the continued clinical development of its product candidates and for other general corporate purposes.

The Company had an accumulated deficit of approximately $471.7 million at March 31, 2015 and anticipates incurring losses through the fiscal year ending December 31, 2015 and beyond. The Company has not yet generated commercial sales revenue and has been able to fund its operating losses to date through the sale of its redeemable convertible preferred stock, issuance of convertible notes, net proceeds from its initial public offering (“IPO”) and subsequent stock offerings, payments from partners during the terms of the collaboration agreements and other financing arrangements. The Company believes that its existing cash and cash equivalents and short-term investments will be sufficient to fund the current operating plan into the second half of 2016.