trending Market Intelligence /marketintelligence/en/news-insights/trending/7vgr_kw9ubfszfo3_e296q2 content esgSubNav
In This List

Amicus Therapeutics' drug for Fabry disease approved in Japan


Insight Weekly: PE firms shift strategies; bank earnings kick off; bankruptcies plummet


Insight Weekly: Stocks limp into 2023; GCC banks set for rebound; deep-sea mining faces pushback


Infographic: The Big Picture 2023 Sustainability Outlook


The Big Picture for 2023: Assessing Investment Trends and the Impact of Investor Activism

Amicus Therapeutics' drug for Fabry disease approved in Japan

Amicus Therapeutics Inc. said Japan's Ministry of Health, Labour and Welfare approved Galafold to treat Fabry disease, a rare genetic disorder.

The approval covers 123-milligram Galafold capsules for patients 16 years and older with Fabry disease with a mutation that makes it amenable to treatment.

The regulator approved the therapy as part of a priority review program for an orphan drug. Amicus Therapeutics expects to launch the drug, also called migalastat, within the coming months.

The company said Galafold is the first oral precision medicine for Fabry disease in Japan.

Fabry disease is a progressive inherited lysosomal storage disorder that results in pain, kidney failure, heart disease and stroke. About 850 people in Japan have Fabry disease, Amicus said.

Cranbury, N.J.-based Amicus Therapeutics is seeking approval for Galafold in the U.S. after securing a recommendation from U.K. regulators in 2017.