Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. "We begin 2017 in an excellent position to develop and deliver great medicines for patients and to create significant shareholder value. For this year, we are laser focused on five key strategic priorities: 1) advancing the international Galafold launch, 2) completing our regulatory submission for migalastat in Japan, 3) establishing our novel Pompe treatment paradigm ATB200/AT2221 as a highly differentiated therapy, 4) successfully completing our Phase 3 clinical study in patients with epidermolysis bullosa, and 5) maintaining our financing strength. With one commercial-stage medicine and two medicines in mid- and late-stage clinical development, as well as a biologics platform for future growth, our vision is to become a leading global biotechnology company focused on delivering meaningful benefits for patients with devastating rare diseases." Key 2016 Accomplishments Galafold(TM) (migalastat) full approval in European Union (EU) - first precision medicine and first oral treatment for Fabry disease received a broad label for patients 16 years and older with a confirmed diagnosis of Fabry disease and who have an amenable genetic mutation (estimated 35%-50% of the global Fabry population) Galafold international launch success - initial launch exceeding expectations with 61 patients on reimbursed Galafold (commercial or expanded access programs, or EAPs) as of December 31, 2016 ATB200/AT2221 positive preliminary data from Pompe clinical study - compelling data on safety and pharmacokinetic (PK) profile in addition to biomarkers of muscle damage set important foundation for differentiating ATB200/AT2221 from any http://www.forbes.com/sites/elyrazin/2017/01/04/how-business-owners-can-refinance-properties-with-government-backed-loans-under-linda-mcmahons-sba/ other approach Enrollment near complete in Phase 3 epidermolysis bullosa (EB) Study - significant momentum with sites now active in the U.S., Europe, and Australia Balance sheet strengthened - cash, cash equivalents, and marketable securities totaled $331 million at December 31, 2016 Mr. Crowley will discuss Amicus' corporate objectives and key milestones in a presentation at the 35th Annual J.P. Morgan Healthcare Conference on Tuesday, January 10, 2017 at 8:30 a.m. PT (11:30 a.m. ET). A live webcast of the presentation can be accessed through the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/events.cfm , and will be archived for 90 days.
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