By Marie Rosenthal
The FDA approved migalastat (Galafold, Amicus Therapeutics US), the first oral medication for the treatment of adults with Fabry disease and the first drug approved for the disease in more than 15 years.
The drug is indicated for adults with Fabry disease who have a genetic mutation determined to be amenable to treatment with migalastat based on in vitro assay results. Fabry disease is a rare and serious genetic disease that results from buildup of a fat called