FDA approves a new oral treatment for Fabry disease

11 August 2018

The US Food and Drug Administration on Friday granted accelerated approval for Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease.

The drug, from US biotech firm Amicus Therapeutics (Nasdaq: FOLD), is indicated for adults with Fabry disease who have a genetic mutation determined to be responsive (“amenable”) to treatment with Galafold based on laboratory data.

Fabry disease is a rare and serious genetic disease that results from build-up of a type of fat called globotriaosylceramide (GL-3) in blood vessels, the kidneys, the heart, the nerves and other organs.

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