A European Medicines Agency (EMA) committee is beginning a review of Sarepta Therapeutics‘ request for conditional approval of eteplirsen as a treatment for Duchenne muscular dystrophy (DMD) patients with a confirmed mutation of the dystrophin gene that can be overcome by exon 51 skipping.
The Committee for Human Medicinal Products (CHMP) validated a Marketing Authorization application (MAA) that Sarepta submitted for conditional approval of eteplirsen in the European Union. The validation confirms that the submission is complete, and a formal review process by CHMP will start.
Assuming a positive CHMP opinion and standard assessment timing, which is 210 days plus time for Sarepta to respond to questions, a final decision from the European Commission regarding eteplirsen is anticipated in late 2017.
“Around the world, there are many patients living with DMD who do not have access to a medicine that treats the underlying cause of the disease,” Edward Kaye, Sarepta’s chief executive officer, said in a recent press release. “The validation of the MAA is the next step toward our goal of providing potential therapies to more patients with Duchenne in Europe.”
Under the brand name Exondys 51, eteplirsen was approval by the U.S. Food and Drug and Administration (FDA) in September 2016, making it the first FDA-approved therapy specifically indicated to treat Duchenne MD. But Exondys 51’s development and approval process was a prolonged one. The FDA decision also followed a campaign by muscular dystrophy advocacy groups urging access to a therapy offering some level of clinical improvement for a disease with no therapeutic options.
DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. The underlying cause of the disease is an error in the gene that codes for dystrophin, an essential protein involved in the muscle function. An estimated 13 percent of people with the DMD have mutations that may be addressed by exon 51 skipping.
Eteplirsen addresses DMD’s underlying cause by restoring the dystrophin messenger RNA (mRNA) reading frame, to enable the production of a shorter, functional form of the dystrophin protein. Data from clinical studies conducted in DMD patients demonstrated that the drug was safe and well-tolerated, and that treatment results in measurable production of the dystrophin protein. Promoting the synthesis of a shorter dystrophin protein may slow the decline of ambulation and mobility seen in DMD patients.