Oral Fabry Drug Clears CHMP, Amicus Sets Up 'Searchable Mutation' Website

Amicus Therapeutics Inc., which is having a roller coaster ride with its oral Fabry disease treatment, is now in touching distance of getting the drug approved in Europe. The EMA's advisory committee, CHMP, gave migalastat (Galafold) a positive recommendation on April 1, 2016 for the treatment of Fabry disease in all patients who have amenable genetic mutations.