Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced that the National Institute for Health and Care Excellence (NICE) Highly Specialised Technologies Evaluation Committee (EC) has issued a positive final evaluation determination (FED) for reimbursed patient access to Galafold™ (migalastat). NICE has recommended the commissioning of Galafold for use within the National Health Service (NHS) in England 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 (alpha-galactosidase A deficiency) and who have an amenable mutation. The Company expects to launch Galafold in England, Wales and Northern Ireland in early 2017.
"Today marks a significant step forward for the Fabry community in the UK," stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. "Galafold will be the first oral treatment as well as the first precision medicine in England, Wales and Northern Ireland for Fabry patients 16 years and older who have an amenable mutation. I would like to sincerely thank NICE, as well as the leading physician experts, patients, and advocacy groups who have collaborated with us throughout this evaluation process. On the heels of significant momentum for our international Galafold launch, we are moving rapidly to make Galafold available on a commercial basis for patients in the UK."
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).
"The MPS Society, supporting over 500 Fabry patients in the UK is pleased to support the reimbursement of Galafold in England," said Christine Lavery, Group Chief Executive and President of the Fabry International Network (FIN). "People in England with Fabry disease who have an amenable mutation will soon have the opportunity to be prescribed an important new oral treatment option. We look forward to the availability of Galafold for patients in England and value Amicus' ongoing collaboration with the Fabry community."
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.
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.<...
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