Sarepta's Eteplirsen Receives EMA Validation for Authorization Application

Andrew Black

Some news out of Europe for the Duchenne community as the European Medicines Agency (EMA) validated the Marketing Authorization application (MMA) for Eteplirsen. Another step closer to having a Duchenne muscular dystrophy (DMD) treatment in Europe. 
Eteplirsen’s recent validation secures that the submission is accepted and starts the formal review process by the EMA’s Committee for Human Medicinal Products (CHMP). This usually takes about 210 days.
Eteplirsen received US FDA accelerated approval for DMD patients in September and was approved under the accelerated approval pathway. It costs cost around $300,000 annually for patients. 
The FDA concluded that the data submitted demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in some patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping.

About Eteplirsen 

Eteplirsen is designed to enable RNA to skip over the part of the DNA with a problematic mutation, enabling a functional (though shorter) dystrophin protein to be produced. Specifically, the drug skips a portion of the DNA known as exon 51. About 13% of the 35,000 DMD patients in the US and Europe have a mutation that might theoretically respond to a drug such as eteplirsen.

About Duchenne muscular dystrophy

Duchenne muscular dystrophy is caused by lack of a functional dystrophin protein, a protein that helps keep muscle cells intact. Patients with progressive muscle disorder experience symptoms in early childhood, losing the ability to walk as early as age 10. These patients, mostly boys, experience life-threatening heart and lung complications in their late teens and twenties.
There are many subsets of the Duchenne population based on the type of mutation found in the dystrophin gene. There are currently no drugs approved in the US to directly treat any of these groups.
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