Canadian Drug Expert Committee Grants Positive Recommendation for Reimbursement of Galafold™ (migalastat) for Fabry Disease in Canada

Philadelphia, Pennsylvania, UNITED STATES

CRANBURY, N.J., Jan. 29, 2018 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) announced that the Common Drug Review (CDR) has posted the Canadian Drug Expert Committee (CDEC)’s positive recommendation of Galafold™ (migalastat) for listing with provincial drug formularies. CDEC recommends reimbursement of Galafold as indicated in the Canadian label, for long-term treatment of adults with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation. Following the positive CDEC recommendation, Amicus plans to pursue discussions with the mandated authorities in order to make Galafold available to Canadian patients.

"The positive recommendation for Canadian reimbursement reflects an important endorsement of the Galafold value proposition as an oral precision medicine for people living with Fabry disease who have amenable mutations,” stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “I would like to thank the Canadian health authorities, as well as leading physician experts and patient groups who provided critical input into this reimbursement decision. We look forward to finalizing our pricing discussions as we prepare to launch Galafold in Canada.”

CDEC considered the following information to reach a positive recommendation: a systematic review of randomized controlled trials and pivotal studies of migalastat and the manufacturer’s pharmacoeconomic submission. The committee also considered input from a clinical expert with experience treating patients with Fabry disease, and patient group-submitted information about outcomes and issues important to patients and caregivers who are affected by Fabry disease.

"Following the positive CDEC recommendation, Fabry patients in Canada who have amenable mutations are one step closer to accessing this important oral precision medicine,” said Dr. Daniel Bichet, Full Professor and Section Head, Renal Function & Transport Physiology, University of Montreal, and Principal Investigator for Canada in the Galafold clinical studies. “Approximately 270 mutations have been identified as amenable to this chaperone therapy in Canada, providing an opportunity for many patients to potentially benefit from this new treatment."

Julia Alton, Executive Director of the Canadian Fabry Association, stated, “I am pleased that Fabry patients in Canada who have amenable mutations will soon have access to a new and differentiated treatment option. We appreciate Amicus’ strong partnership with the Fabry community and we commend CDEC for considering the patient perspective in the reimbursement review process.”

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).

About Galafold™ and Amenable Mutations
Galafold™ (migalastat) is a first-in-class chaperone therapy approved in Canada 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 1,000 known GLA mutations as “amenable” or “not amenable” to treatment with Galafold. The Canadian label includes 270 GLA mutations that have been identified and determined to be amenable in Canada based on the Galafold Amenability Assay.

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

Important Canadian 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 non-amenable mutation.

  • Clinical data supporting the effectiveness of Galafold for the treatment of Fabry disease patients with amenable mutations are limited. In clinical trials, individual response to Galafold treatment varied considerably among patients with amenable mutations. Patients should be assessed for treatment response or failure when initiating Galafold, and monitored periodically thereafter (every 6 months or more frequently) throughout the treatment.
  • Galafold is not recommended for use in patients with a non-amenable mutation. Galafold may result in a net loss of α-Gal A activity in patients with non-amenable mutations, potentially worsening the disease condition.
  • Galafold is not intended for concomitant use with enzyme replacement therapy.
  • The patient should be advised to carefully adhere to the recommended dosing regimen of Galafold [one 123 mg migalastat capsules every other day (QOD)]. A higher dose or shorter dosing interval may result in a loss of efficacy, potentially worsening the disease condition.
  • Galafold should not be used in patients with severe renal insufficiency, defined as having an estimated glomerular filtration rate (eGFR) less than 30 mL/min/1.73m2 , due to a significant increase in the exposure to migalastat and prolonged half-life of migalastat. This may result in a net loss of α-Gal A activity, potentially worsening the disease condition.
  • The safety and efficacy of Galafold in pediatric patients have not been established.
  • No dosage adjustments are required in patients with hepatic impairment or in the elderly population.
  • Galafold should not be used by pregnant women and is not recommended in women of childbearing potential not using contraception.
  • While taking Galafold, effective birth control should be used. Galafold should not be used in breast-feeding women. It is not known whether Galafold is excreted in human milk.
  • Galafold is contraindicated for use in patients who are hypersensitive to this drug or to any ingredient in the formulation or component of the container.
  • OVERDOSE: General medical care is recommended in the case of Galafold overdose.
  • The most common adverse reaction reported was headache. For a complete list of adverse reactions, please review the Canadian Product Monograph.
  • Call your doctor for medical advice about side effects.   

For further important safety information for Galafold, including the indications,  method of administration, special warnings, drug interactions and adverse drug reactions, please see the Canadian Prescribing Information for Galafold available from the Health Canada website here.

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, patient-centric biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. The cornerstone of the Amicus portfolio is migalastat, an oral precision medicine for people living with Fabry disease who have amenable genetic mutations. Migalastat is currently approved under the trade name Galafold™ in the European Union, with additional approvals granted and pending in several geographies. The future value driver of the Amicus pipeline is ATB200/AT2221, a novel, late-stage, potential best-in-class treatment paradigm for Pompe disease. The Company is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. 

Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. 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 commercialization of Galafold, 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 we may not be successful in commercializing Galafold in Canada, Europe and other geographies or our other product candidates if and when approved. 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.

Amicus Therapeutics
Sara Pellegrino, IRC
Senior Director, Investor Relations
(609) 662-5044

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