Week 2

January 2016; The FDA has issued a Complete Response letter to BioMarin’s New Drug Application (NDA) for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping. A Complete Response letter indicate that the FDA has reviewed the drug application and concluded that the standard of substantial evidence of effectiveness has not been met. Have a look at a movie clip from the Washington Post about this dramatic event;