FDA grants accelerated approval to first targeted treatment for rare Duchenne Muscular Dystrophy (DMD) mutation

FDA grants accelerated approval to first targeted treatment for rare Duchenne Muscular Dystrophy (DMD) mutation

The U.S. Food and Drug Administration 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 most common side effects reported by participants receiving Vyondys 53 in clinical studies were headache, fever (pyrexia), cough, vomiting, abdominal pain, cold symptoms (nasopharyngitis) and nausea. Hypersensitivity reactions, including rash, fever, itching, hives, skin irritation (dermatitis) and skin peeling (exfoliation), have occurred in patients who were treated with Vyondys 53. Additionally, renal toxicity was observed in animals who received golodirsen. Although renal toxicity was not observed in the clinical studies with Vyondys 53, renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Renal function should be monitored in patients taking Vyondys 53.

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