Australian TGA approves Amicus' Galafold to treat Fabry disease

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 drug development, and high quality therapies for the Fabry community."

The European Commission granted full approval for Galafold in May 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. Marketing applications have also been approved in several countries outside the EU, including Switzerland, Israel, and now Australia.

Galafold (migalastat) is a first-in-class chaperone therapy approved in Australia 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 Australian label includes 331 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.

Amicus expects to submit additional updates to the Australian label as additional GLA mutations are identified and tested in the Galafold Amenability Assay.

Australian tga, amicus' galafold to treat fabry disease