Amicus Therapeutics has received the Australian Therapeutic Goods Administration (TGA) approval for Galafold (Migalastat) to treat patients with Fabry disease (alpha-galactosidase A deficiency) in the country.
Fabry disease is a rare genetic 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.
Amicus Therapeutics chairman and chief executive officer John Crowley said: “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.”
The oral precision medicine Galafold is a first-in-class chaperone treatment used as a monotherapy for Fabry disease in patients aged 16 and older and with amenable mutations.
The treatment helps stabilise the body's own dysfunctional enzyme so that it can clear the accumulation of disease substrate in patients who have amenable mutations.
On receiving the TGA approval, the US biopharmaceutical company continues to work in collaboration with the Australian reimbursement authorities to ensure that Fabry disease patients in the country have timely access to Galafold.
Crowley added: “Following our initial European Union (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.”
Image: Alpha galactosidase – the deficient protein Fabry disease. Photo: courtesy of ProteinBoxBot at English Wikipedia.