Amicus Therapeutics Inc (NASDAQ:FOLD) an international biotechnology company with its focus on discovering, delivering and developing unique latest drugs for rare metabolic ailments, provided its full-year strategic outlook for 2018.
In 2017, Amicus surpassed “Target 300” goal with over 310 people cured with reimbursed Galafold™ oral precision drug for Fabry disease at year-close 2017. Full-year 2017 revenue for Galafold totaled around $36 million. The company closed international regulatory filings for migalastat in Japan, the U.S., and other major geographies. It established vital clinical proof-of-concept for unique, highly distinguished Pompe treatment plan ATB200/AT2221 on functional, safety outcomes and major disease biomarkers.
Amicus successfully scaled manufacturing of Pompe biologic engineering batches done at commercial scale with capacity plans to confirm that it serves entire Pompe population as quickly as possible. The company strengthened balance sheet as total cash/cash equivalents and marketable securities came at $359 million as of the close of December 31, 2017.
John F. Crowley, the CEO and Chairman of Amicus Therapeutics, reported that during 2017 they continued to build a major global rare ailment biotech company while developing their vision to increase the impact of their medicines for people struggling with rare diseases.
They surpassed their EU release and regulatory goals for their Fabry precision drug Galafold, and they posted clinical data from their Pompe clinical trial that lays the base for a prospective new treatment paradigm for the respective muscle problem. Throughout 2018 they are set to create considerable additional value for shareholders and patients across their key programs in Pompe and Fabry, and through their focused commitment to expanding and advancing a pipeline of novel drugs for rare metabolic ailments.
Amicus focus will be on five major strategic priorities in this year, which includes double international revenue for Galafold; get approvals for migalastat in the U.S. and Japan; get clinical, regulatory and manufacturing milestones to develop ATB200/AT2221 toward international regulatory submissions; expand and develop preclinical pipeline to confirm at least one new clinical plan in 2019; and maintain an impressive balance sheet.