Amicus Therapeutics Announces Approval of Galafold® (Migalastat) for Fabry Disease in Japan

Amicus Therapeutics (Nasdaq:FOLD) announced today that Japan’s Ministry of Health, Labour and Welfare has approved the oral small molecule pharmacological chaperone Galafold capsules 123mg (migalastat) for treatment of patients aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.

As quoted in the press release:

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “The Japanese approval for Galafold is a major step forward for more than 800 people currently known to be living with Fabry disease in Japan. We believe a significant portion of these Fabry patients have amenable mutations that are suitable for treatment with this differentiated precision oral therapy. I would like to highlight the tremendous collaboration among our Amicus employees, Japanese regulators, and the Fabry community, in particular those physicians and patients who participated in the clinical studies of Galafold and their families who made this approval possible. Japan is very important to our patient-focused vision to provide Galafold to Fabry patients with amenable mutations throughout the world as soon as possible. And now that Amicus has established a strong presence in Japan, we hope that the upcoming Galafold launch will be the first of many future opportunities to deliver new medicines for people living with rare metabolic diseases in Japan.”

Click here to read the full press release.