Rare Daily Staff
The Committee for Medicinal Products for Human Use of the European Medicines Agency confirmed its May 31, 2018 negative opinion for a Conditional Marketing Application for Sarepta Therapeutics’ eteplirsen, the company’s Duchenne muscular dystrophy amenable to exon 51 skipping.
The company expects the European Commission to adopt the CHMP opinion by the end of 2018.
DMD is a rare, progressive, and fatal muscle disorder caused by the lack of functional dystrophin protein. Patients with the condition lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties. About 13 percent of DMD patients have genetic mutations amenable to exon 51 skipping.
“While largely anticipated, we are disappointed with the outcome of the CHMP re-examination and firmly believe that eteplirsen should be made available to patients in Europe, as it is in the United States,” stated Doug Ingram, president and CEO of Sarepta.
Ingram said the company was encouraged by the “openness” of the discussion with CHMP and its willingness to engage on different approaches to provide additional data to support an eventual approval in Europe.
Based on those discussions, Sarepta will work to explore a potential path forward. The company said it will be seeking follow-up scientific advice in 2019 to explore the approach to bring eteplirsen to Europe.
September 24, 2018
Photo: Doug Ingram, president and CEO of Sarepta