FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation
The U.S. Food and Drug Administration today granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. It is estimated that about 8 percent of patients with DMD have this mutation. " The FDA recognises the urgent need for new medical treatments for serious neurological disorders and we have a long-standing commitment to working with researchers, drug companies and patients to facilitate the development and approval of treatments for rare diseases. With today's accelerated approval, patients with Duchenne - a rare and devastating disease - who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping will now have available the first treatment targeted specifically for this disease subtype ," said Billy Dunn, M.D., acting director of the Office of Neuroscience in the FDA's Centre for Drug Evaluat...