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Amicus Therapeutics Presents Updated Patient Screening Profiles From Phase 3 Fabry Study

Study 011 is a randomized study with a 6-month, double-blind, placebo-controlled primary treatment period and a 6-month, open-label follow-up period in which all subjects receive treatment with migalastat HCl. The primary endpoint in Study 011 is a change in interstitial capillary GL-3 as measured in kidney biopsy at 6 months versus baseline.  The 6-month primary treatment period in Study 011 was completed in the second quarter 2012 and the 6-month follow-up period is expected to complete in the fourth quarter 2012. Amicus and GSK expect to unblind and analyze data from the primary 6-month treatment arm during the fourth quarter 2012. Both companies remain blinded to all results at this time.

John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, "Screening results from Study 011 suggest that migalastat HCl monotherapy may address a substantial portion of the Fabry population on the basis of genotype. In addition, these screening results may provide the Fabry patient and physician community with valuable information on a wide variety of different mutations, including newly discovered mutations."

1.     Migalastat HCl, an Investigational Pharmacological Chaperone Therapy: Screening Results from FACETS, a Phase 3 Study in Male and Female Fabry Patients. – Nicholls, K., Castelli J., Winkler R., Sitaraman, S., Benjamin E., Wu, X., Duke C., Boudes, P., on Behalf of FACETS Study AT1001-011 Principal Investigators.

About Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of developing therapies for rare diseases. The Company is developing orally-administered, small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of human genetic diseases. Amicus' late-stage programs for lysosomal storage disorders include migalastat HCl monotherapy in Phase 3 for Fabry disease; migalastat HCl co-administered with enzyme replacement therapy (ERT) in Phase 2 for Fabry disease; and AT2220 co-administered with ERT in Phase 2 for Pompe disease.

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