Amicus Therapeutics’ Galafold Approved in Europe for Fabry Disease
The European Commission has extended its marketing authorization for Amicus Therapeutics’ Galafold (migalastat) for treatment of the rare inherited disorder, Fabry disease, to include adolescent patients age 12 to 15.
The approved indication is for adolescents who weigh at least 45 kilograms and who have a confirmed diagnosis of Fabry disease and certain mutations in the gene that encodes the alpha-galactosidase A enzyme. The disorder can lead to irreversible organ damage, kidney failure, heart disease and stroke.
Galafold is already approved in the EU, the U.S. and elsewhere for adults who have certain variants of the mutated gene.