15 August 2017 - First Amicus medicine and first oral precision medicine for Fabry disease in Australia.

Amicus Therapeutics 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 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) in the lysosome due to a dysfunctional or deficient enzyme. Galafold works by stabilising 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).

Read Amicus Therapeutics press release