Amicus Therapeutics
Mar 8, 2017
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Amicus Therapeutics Launches Galafold™ (Migalastat) for Treatment of Fabry Disease in Italy

Ministry of Health Publishes Final Guidance Covering Reimbursement of Galafold for Fabry Disease

CRANBURY, N.J. and MILAN, Italy, March 08, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, has commenced the commercial launch of the precision medicine Galafold in Italy following the final publication of reimbursement guidelines by the Ministry of Health (Ministero della Salute). Galafold is now reimbursed in Italy as a therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.

"The commercial launch of Galafold in Italy is a significant milestone for the Fabry community," stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. "All Fabry patients in Italy 16 years and older who have an amenable mutation will now have access to the first oral treatment as well as the first precision medicine for their Fabry disease. We believe that our successful pricing and reimbursement in Italy is a testament to the significant value of our oral precision medicine, and reflects our growing momentum with the country-by-country processes to launch Galafold throughout the EU."

Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. As a precision medicine, Galafold is designed to restore alpha-Gal A activity in patients who have amenable mutations (an estimated 35% to 50% of the Fabry population).

Dr. Iacopo Olivotto, manager of the Cardiomyopathy Unit of the Careggi Hospital in Florence, center for Fabry disease stated, "As an active site for the ATTRACT study, we are particularly pleased that Galafold will be available for Fabry patients in Italy. When there are more treatments available on the market for a rare disease, this automatically leads to an increase in the attention given to the disease and awareness surrounding it. Fabry disease is a virtuous example of how targeted treatments, based on a solid rationale, may improve treatment outcomes for patients"

"Every time a new treatment opportunity is added to those available it is a day of celebration for patients," said Stefania Tobaldini, President of the Associazione Italiana Anderson Fabry Onlus (AIAF). "Today our patient association is particularly grateful to scientific research as a new opportunity for treatment is available for the first time via oral administration. As of tomorrow, there will be a new task for the specialists who are following us on our complex paths to diagnosis and treatment; that of informing us and accompanying us in the exploration of Galafold as a new treatment option in Italy for patients with Fabry disease who have an amenable mutation."

The European Commission granted full approval for Galafold on May 30, 2016, as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation.

About Galafold™ and Amenable Mutations
Galafold™ (migalastat) is a first-in-class chaperone therapy approved in the EU as a monotherapy for Fabry disease in patients with amenable mutations. Galafold works by stabilizing the body's own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations.  A proprietary in vitro assay (Galafold Amenability Assay) was used to classify more than 800 known GLA mutations as "amenable" or "not amenable" to treatment with Galafold. The current EU label includes 313 GLA mutations that have been identified and determined to be amenable based on the Galafold Amenability Assay, which represent between 35% and 50% of the currently diagnosed Fabry population.

Healthcare providers in the EU may access the website www.galafoldamenabilitytable.com to quickly and accurately identify which mutations are categorized as "amenable" or "not amenable" to Galafold. Amicus expects to submit updates to the label as additional GLA mutations are identified and tested in the Galafold Amenability Assay.

Important Safety Information
Treatment with GALAFOLD should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. GALAFOLD is not recommended for use in patients with a nonamenable mutation.

For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.

About Fabry Disease
Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease, and stroke. The symptoms can be severe, differ from patient to patient, and begin at an early age. All Fabry disease is progressive and may lead to organ damage regardless of the time of symptom onset.

About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus' lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.

Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, financing plans, and the projected cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we 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 in particular 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 our business, including, without limitation: the potential that results of clinical or preclinical 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, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our 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, 2016. 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 we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.

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