Posted by Michael Wonder on 10 Aug 2018
FDA approves new treatment for a rare genetic disorder, Fabry disease
10 August 2018 - The U.S. FDA today approved Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease.
The drug is indicated for adults with Fabry disease who have a genetic mutation determined to be responsive to treatment with Galafold based on laboratory data. Fabry disease is a rare and serious genetic disease that results from buildup of a type of fat called globotriaosylceramide in blood vessels, the kidneys, the heart, the nerves and other organs.
The efficacy of Galafold was demonstrated in a six-month, placebo-controlled clinical trial in 45 adults with Fabry disease.
