Amicus Therapeutics Receives Marketing Authorization for Galafold® (migalastat) for Fabry Disease in Argentina
First Oral Precision Medicine Approved for People Living with Fabry Disease with an Amenable Mutation in
First Amicus Regulatory Approval in
Amicus is working with a local partner, Pint Pharma, to complete the requirements to launch Galafold® in the coming months.
“The approval of Galafold® in
The Argentinian label for Galafold® includes over 350 GLA mutations that have been identified and determined to be amenable based on the Galafold Amenability Assay. It is estimated that between 35% and 50% of people diagnosed with Fabry disease may have an amenable GLA mutation.
Hernan Amartino, MD, Head of the
In addition to
About Galafold® and Amenable Mutations
Fabry disease is a rare genetic disease and potentially life-threatening condition caused by the accumulation of disease substrate (globotriaosylceramide, GL-3) in the lysosome due to a dysfunctional or deficient enzyme. In patients with an amenable mutation, Galafold® (migalastat) works by stabilizing the body's own dysfunctional enzyme, so it can enter the lysosome. Once inside the lysosome, Galafold® detaches from the enzyme, allowing it to break down substrate. An amenable mutation is one that results in an enzyme that can be stabilized by Galafold®. Amenability is determined based on a proprietary in vitro assay (Galafold Amenability Assay).
Healthcare providers in
Important Argentine Safety Information
Treatment with Galafold® should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. Galafold® is not indicated for use in patients with a non-amenable mutation.
- The efficacy and the safety of concomitant use with enzyme replacement therapy has not been established. Galafold® is not recommended for use in patients with Fabry disease who have severe renal impairment. The safety and efficacy of Galafold® in low birth weight infants, neonates, nursing infants, infants and children have not yet been established.
- Migalastat exposure is affected by food, therefore it should not be taken within 2 hours before and after food.
- Patients should be observed carefully, and caution should be taken in the administration in the elderly population.
- If you are pregnant, think you may be pregnant, or are planning to have a baby, do not take this medicine until you have checked with your doctor, pharmacist, or nurse, and consider the treatment only in case that the benefit from migalastat is judged to exceed the risk during pregnancy. Nursing mothers should be instructed not to breast-feed if they are taking migalastat or to discontinue migalastat if they do breast-feed
- Contraindications to Galafold® include hypersensitivity to the active substance or to any of the excipients listed in the PRESCRIBING INFORMATION.
- Patients should be monitored based on their course including renal and cardiac function and clinical laboratory test during migalastat treatment. In case no effect is observed in the migalastat treatment, changing treatment should be considered.
- OVERDOSE: Headache and dizziness were the most common adverse reactions reported at doses of migalastat of up to 1250 mg and 2000 mg, respectively, administrated in healthy subjects in the overseas clinical studies.
- The most common adverse reaction reported was headache, which was experienced by approximately 10% of patients who received Galafold®. For a complete list of adverse reactions, please review the Argentinian Galafold® package insert.
- Call your doctor for medical advice about side effects.
For further important safety information for Galafold®, including dosage and administration, precautions, drug interactions and adverse drug reactions, please see the
About Fabry Disease
Fabry disease is an inherited lysosomal disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which results from 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 heart, kidneys, and skin. Accumulation of GL-3 and progressive deterioration of organ function is believed to lead to the morbidity and mortality of Fabry disease. The symptoms can be severe, differ from person to person, and begin at an early age.
About Pint Pharma
Forward Looking Statement
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to approval and commercialization of Galafold® in Argentina. 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, 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 the
Vice President, Investor Relations & Corporate Communications
Director, Corporate Communications
Source: Amicus Therapeutics, Inc.