Relying upon Scientific Advisory Group input, Sarepta will seek further scientific advice from European Medicines Agency on a possible path to bring eteplirsen to patients in Europe
CAMBRIDGE, Mass., September 21, 2018 (GLOBE NEWSWIRE) – Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has confirmed its 31 May 2018 negative opinion for a Conditional Marketing Application for eteplirsen. Eteplirsen is designed to treat approximately 13% of the Duchenne muscular dystrophy community who 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 chief executive officer, Sarepta Therapeutics.
Mr. Ingram continued, “We were, however, encouraged by the openness of discussion with the SAG and CHMP and their 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 that balances the needs of patients and their families to avoid lengthy and unnecessarily burdensome trials with those of European Regulators for additional supportive data consistent with existing European regulations. We will be seeking follow up Scientific Advice in 2019 in order to explore the approach to bring eteplirsen to Europe.”
The Company expects the European Commission (EC) to adopt the CHMP opinion by year-end 2018.