December 2016

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage developer of innovative RNA-targeted therapeutics, today announced that the European Medicines Agency(EMA) validated the previously submitted Marketing Authorization application (MAA) for eteplirsen to treat Duchenne muscular dystrophy amenable to exon 51 skipping. Sarepta is seeking conditional approval of eteplirsen in the EU through the centralized procedure. Validation of the MAA confirms that the submission is accepted and starts the formal review process by the EMA’s Committee for Human Medicinal Products (CHMP). The standard review period is 210 days (plus additional time for applicant to respond to questions from the agency).

“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,” said Edward Kaye, Sarepta’s chief executive officer. “The validation of the MAA is the next step toward our goal of providing potential therapies to more patients with Duchenne in Europe.”

About Eteplirsen
Eteplirsen uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical studies of eteplirsen in a small number of DMD patients have demonstrated a consistent safety and tolerability profile. The pivotal trials were not designed to evaluate long-term safety and a clinical benefit of eteplirsen has not been established.

About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates. For more information, please visit us at www.sarepta.com.

Source: Sarepta Therapeutics, Inc.

Media and Investors:
Sarepta Therapeutics, Inc.
Ian Estepan, 617-274-4052
iestepan@sarepta.com
or
W2O Group
Brian Reid, 212-257-6725
breid@w2ogroup.com