Amicus Therapeutics Announces Approval for Galafold™ (Migalastat) for Treatment of Fabry Disease in Canada
"The approval of Galafold represents a significant step forward for the Canadian Fabry community," stated
"Following more than a decade of experience treating Fabry patients with Galafold in the clinical setting, I am pleased that Fabry patients in
About Galafold™ and Amenable Mutations
Galafold™ (migalastat) is a first-in-class chaperone therapy approved in
Healthcare providers in
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 GALAFOLDTM 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
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 Gb
3). 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
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to clinical development of our product candidates, the prospects and timing of the potential regulatory and pricing approval of our product candidates. 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 and pricing
authorities 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 we may not be successful in commercializing Galafold in
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Investors/Media:
Senior Director, Investor Relations
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(609) 662-5044
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