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February 25, 2021Amondys 45 Approved to Treat Duchenne Muscular Dystrophy
February 25, 2021 – The U.S. FDA has approved Amondys 45TM (casimersen), manufactured by Sarepta Therapeutics, to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene amenable to exon 53 skipping.
DMD is a rare genetic condition that causes muscle loss and weakness. It occurs when a mutation alters the production of dystrophin, a protein needed to maintain the health and integrity of muscle cells. Symptoms can manifest as early as age two. Children with DMD may have difficulty with everyday activities like walking and jumping and can also develop a curved spine. As the disease progresses, it can interfere with heart and lung function.
In a clinical study, patients who received Amondys 45 experienced statistically significant increases in dystrophin production in their skeletal muscles. Recommended dosing is 30mg/kg of the patient’s body weight, administered via intravenous infusion over the course of 35-60 minutes once weekly.
The FDA granted Amondys 45 accelerated approval, meaning that continued approval may be contingent on verification of clinical benefit in confirmatory trials. The product has launched at a wholesale acquisition cost (WAC) of $1,600 per 100mg vial.