Successful Pre-Submission Meeting with European Medicines Agency Completed
MAA Submission Anticipated in Mid-2015
CRANBURY, N.J., Dec. 11, 2014 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD ), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced that it has conducted a successful pre-submission meeting with the European Medicines Agency (EMA) to discuss the registration of migalastat HCl ("migalastat") monotherapy for the treatment of Fabry disease. Migalastat is an oral small molecule pharmacological chaperone in development for Fabry patients with amenable mutations. Amicus has begun preparing a marketing authorization application (MAA) that it plans to submit to the EMA in the middle of 2015 under the Centralized Procedure.
"The submission of our marketing application in Europe will be an important milestone in our global strategy to get migalastat approved for Fabry patients with amenable mutations as quickly as possible," said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics. "We believe significant unmet need still exists in the treatment of Fabry disease and that migalastat could become a very important, differentiated oral therapy. We also look forward to meeting with the FDA in the first quarter of 2015 to discuss the U.S. regulatory path."
As previously reported , migalastat successfully met both co-primary endpoints of comparability to enzyme replacement therapy (ERT) on both key measures of kidney function in a Phase 3 global registration study ( Study 012 ) that compared migalastat to standard-of-care ERTs in Fabry patients with amenable mutations. Study 012 was designed based on feedback from the EMA. The Company has also previously announced positive 12- and 24-month results from a Phase 3 global registration study ( Study 011 ) that demonstrated the benefit of migalastat compared to placebo in Fabry patients with amenable mutations.
About Study 012
Study 012 was a Phase 3, open-label study that compared oral migalastat to standard-of-care ERTs for Fabry disease (Fabrazyme® and Replagal®). The study enrolled 60 patients (26 males and 34 females) with Fabry disease with amenable mutations in a clinical trial assay who had been treated with ERT for a minimum of 12 months prior to study entry. These patients were randomized 1.5:1 to switch to migalastat (36 patients) or remain on ERT (24 patients) for the primary 18-month treatment period, after which they were eligible to receive migalastat in a 12-month extension phase.
The co-primary outcome measures were the mean annualized changes in estimated glomerular filtration rate (eGFR) and measured (iohexol) GFR (mGFR) assessed by descriptive comparisons of migalastat and ERT over 18 months. Secondary outcome measures included cardiac function assessed by echocardiography, as well as a composite of Fabry-associated clinical events (i.e. renal, cardiac, or cerebrovascular).
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD ) is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company is developing novel, first-in-class treatments for a broad range of human genetic diseases, with a focus on delivering new benefits to individuals with lysosomal storage diseases. Amicus' lead programs include the small molecule pharmacological chaperones migalastat as a monotherapy and in combination with enzyme replacement therapy (ERT) for Fabry disease; and AT2220 (duvoglustat) in combination with ERT for Pompe disease.
Forward-Looking Statements
This press release contains, and the accompanying conference call will contain, "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of Amicus' candidate drug products, the timing and reporting of results from preclinical studies and clinical trials evaluating Amicus' candidate drug products and the projected cash position for the Company. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "potential," "plan," "planned," "targets," "likely," "may," "will," "would," "should" and "could," and similar expressions or words identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. The inclusion of forward-looking statements should not be regarded as a representation by Amicus that any of its plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong. They can be affected by inaccurate assumptions Amicus might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing and outcomes of discussions with regulatory authorities and the potential goals, progress, timing and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in the business of Amicus, including, without limitation: the potential that results of clinical or pre-clinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities may not grant or may delay approval for our product candidates; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we will need additional funding to complete all of our studies and, our dependence on third parties in the conduct of our clinical studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2013. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and Amicus undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
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Investors/Media: Amicus Therapeutics Sara Pellegrino Director, Investor Relations (609) 662-5044 Media: Pure Communications Dan Budwick (973) 271-6085
