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Amicus Therapeutics Announces Approval for Galafold™ (Migalastat) for Treatment of Fabry Disease in Australia

First Amicus Medicine and First Oral Precision Medicine for Fabry Disease in Australia

Broad Label for Fabry Patients with an Amenable Genetic Mutation

CRANBURY, N.J., Aug. 15, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (FOLD) announced that the Australian Therapeutic Goods Administration (TGA) has approved the oral precision medicine Galafold 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. Amicus estimates that approximately 35%-50% of Fabry patients in Australia have an amenable mutation. Following the TGA approval, Amicus is continuing to work with the Australian reimbursement authorities to make Galafold available to Australian patients in a timely manner.

"The approval of Galafold in Australia is a significant step forward for the Fabry community and reflects our commitment to providing the first oral precision medicine for Fabry disease as rapidly as possible to patients throughout the world,” stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “There has been a tremendous amount of momentum for the commercial launch and significant progress with our regulatory submissions and approvals for Galafold. Following our initial EU approval, this is our second approval through an independent submission process. We have also secured approvals in geographies such as Switzerland and Israel that have a regulatory pathway that accepts the EU approval as the basis for submission and review. Our next step in Australia is to navigate the pricing and reimbursement discussions as we continue toward our vision to deliver Galafold to even more patients in more geographies.”

The Australian TGA approval under the Orphan Drug program was based on clinical data from two Phase 3 pivotal studies in both treatment naïve (Study 011, or FACETS) and enzyme replacement therapy (ERT) switch patients (Study 012, or ATTRACT), as well as an ongoing long-term extension study. 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. 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. An amenable mutation is one that is responsive to therapy with Galafold based on a proprietary in vitro assay (Galafold Amenability Assay).

“The Australian approval of Galafold paves the way for the first new Fabry treatment option in more than a decade, and provides a clearly differentiated oral precision medicine option for Fabry patients in Australia who have amenable mutations,” said Megan Fookes, Managing Director of Fabry Australia. “We are grateful for Amicus’ commitment to innovation, patient-focused...