Eteplirsen approved in US for treatment of duchenne muscular dystrophy

Accelerated approval of the drug, which will treat a subset of those living with Duchenne muscular dystrophy, is an important step forward in the development of therapies for neuromuscular diseases and marks an historic achievement for the entire DMD community.

"Today has been a long time in the making," said MDA President and CEO Steven M. Derks. "This is the outcome MDA dreamed of 25 years ago when it was the first to invest in the breakthrough research that led to development of eteplirsen. Throughout this process we have seen the undeniable strength of our community to rally behind MDA's commitment to find treatments for our families. This is an important victory, and we are honored to stand shoulder-to-shoulder with everyone who has fought to make this day a reality."

Approval to market eteplirsen was given to pharmaceutical company Sarepta Therapeutics. Eteplirsen will be the first disease-modifying drug on the market in the United States to treat DMD, and approximately 13 percent of DMD patients potentially may be eligible for treatment. Under the terms of the FDA's accelerated approval, Sarepta must conduct a clinical trial of eteplirsen to confirm clinical benefit.