Duchenne Muscular Dystrophy Rx Approved

The US Food and Drug Administration has approved the first medication to treat Duchenne muscular dystrophy (DMD).

This new medication is Exondys 51 (eteplirsen) injection. 

Exondys 51 has been approved for those who have a particular type of gene mutation. This mutation affects about 13 percent of those with DMD.

DMD, the most common type of muscular dystrophy, is a rare genetic disorder that affects the muscles. To be exact, those with DMD do not have dystrophin, which is a protein that helps keep the muscle cells together. Those with DMD have progressive muscle deterioration and weakness. 

Exondys 51 is available as an injection and is made by Sarepta Therapeutics. 

The most commonly reported side effects included balance disorder and vomiting.

Exondys 51 has been approved under the accelerated approval pathway. Through this process, medications that treat serious or life-threatening diseases are approved, which enables patients to have earlier access to new medications while the company conducts clinical trials that will determine their benefit.

Sarepta Therapeutics is expected to conduct a clinical trial to determine Exondys 51's efficacy. The trial will determine whether this medication will improve motor function.