Exondys 51 approved for Duchenne muscular dystrophy
September 19, 2016 – The US FDA approved Exondys 51™ (eteplirsen) injection under the accelerated approval process. Exondys 51 is the first FDA-approved drug indicated to treat patients who have Duchenne muscular dystrophy (DMD), specifically for patients confirmed to have mutations of dystrophin genes amenable to exon 51 skipping. DMD is a rare genetic disease that affects around 20,000 boys and young men in the United States. In DMD, a mutation in the gene for dystrophin, a muscle protein, causes progressive muscle wasting. Eventually, DMD patients need wheelchairs and ventilators. Currently, few patients live beyond 30 years of age. Exondys 51 is to be administered via an intravenous (IV) infusion at 30mg/kg of body weight, once a week. Sarepta plans on launching Exondys 51 immediately and it will be available in single-use vials of 50mg/mL in either 2mL or 10mL sizes. It’s expected to cost approximately $350,000 annually.
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