Australian approval for Amicus Fabry disease drug

US biotech Amicus Therapeutics (Nasdaq: FOLD) says the Australian Therapeutic Goods Administration (TGA) has approved its Galafold (migalastat) 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 about 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 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 Crowley, chairman and chief executive Officer of Amicus, adding: "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."