Fabry disease is a progressive, inherited lysosomal storage disorder caused by an enzyme deficiency. The disease causes accumulation of specific lipids in tissues including the central nervous system, heart, kidneys, and skin. This abnormal accumulation can lead to debilitating consequences including pain, kidney failure, heart disease, and stroke.
"This Fast Track designation recognizes that Fabry is a serious disease and that migalastat has the potential to address the significant needs faced by this patient community," stated
The Company plans to submit a New Drug Application (NDA) for migalastat in the fourth quarter of 2017. Drugs with Fast Track designation may qualify for accelerated approval and priority review to expedite the
Migalastat works by stabilizing the body's own dysfunctional enzyme, so it can clear the accumulated disease substrate in patients who have amenable mutations (an estimated 35% to 50% of Fabry patients globally). An estimated 3,000 people in the
Outside the EU, migalastat is approved in
About Fast Track Designation1
Fast Track designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions. A drug that is intended to treat a serious condition with nonclinical or clinical data that demonstrate the potential to address unmet medical need may qualify for Fast Track designation. When Fast Track designation is requested later in development, available clinical data should demonstrate the potential to address an unmet medical need. There are opportunities for frequent interactions with the review team for a fast track product. In addition, such a product could be eligible for priority review if supported by clinical data at the time of BLA, NDA, or efficacy supplement submission.
About Galafold™ and Amenable Mutations
Galafold™ (migalastat) is a first-in-class chaperone therapy approved in the
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 additional updates to the EU label as additional GLA mutations are identified and tested in the Galafold Amenability Assay.
EU 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 non-amenable mutation.
- Galafold is not intended for concomitant use with enzyme replacement therapy.
- Galafold is not recommended for use in patients with Fabry disease who have severe renal impairment ( < 30 mL/min/1.73 m2). The safety and efficacy of Galafold in children 0-15 years of age have not yet been established.
- No dosage adjustments are required in patients with hepatic impairment or in the elderly population.
- There is very limited experience with the use of this medicine in pregnant women. 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.
- While taking Galafold, effective birth control should be used. It is not known whether Galafold is excreted in human milk.
- Contraindications to Galafold include hypersensitivity to the active substance or to any of the excipients listed in the PRESCRIBING INFORMATION.
- It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on Galafold or switched to Galafold.
- OVERDOSE: General medical care is recommended in the case of Galafold overdose.
- 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 SUMMARY OF PRODUCT CHARACTERISTICS.
- Call your doctor for medical advice about side effects.
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.
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to the clinical development, regulatory approval pathway, and prospects and timing of regulatory submission and approval of our product candidates for the treatment of Fabry disease. Any express or implied statements contained in this press release that are not statements of historical fact, including interpretation of guidance given by the
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