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BUSINESS
6 Months Ended
Jun. 30, 2017
Accounting Policies [Abstract]  
BUSINESS

1. BUSINESS

Sarepta Therapeutics, Inc. (together with its wholly-owned subsidiaries, “Sarepta” or the “Company”) is a commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases. Applying its proprietary, highly-differentiated and innovative platform technologies, the Company is able to target a broad range of diseases and disorders through distinct RNA-targeted mechanisms of action. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (“DMD”) drug candidates. On September 19, 2016, the United States Food and Drug Administration (“FDA”) granted accelerated approval for EXONDYS 51, indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. EXONDYS 51 is studied in clinical trials under the name of eteplirsen and is marketed in the U.S. under the trademarked name of EXONDYS 51® (eteplirsen) Injection.

In November 2016, the Company submitted a marketing authorization application (“MAA”) for eteplirsen to the European Medicine Agency (“EMA”) and the application was validated in December 2016. The Company requested and received a six-month clock stop of EMA’s review of its MAA for eteplirsen to complete an ADME study required for the Company’s MAA. In addition to working on the ADME study, the Company is using the clock stop period to collect additional data and conduct additional analyses from existing studies with the goal of submitting these to the EMA to additionally support its MAA, assuming the data and information gathered is positive, and to address any EMA questions or requests.  The Company continues to work with the EMA during their review process and anticipate they will complete their review and make a final decision on the approvability of the Company’s MAA for eteplirsen in 2018. The Company also initiated a Managed Access Program (“MAP”) for eteplirsen in certain geographies to treat DMD patients amenable to exon 51 skipping. This MAP (also known as an early/expanded access, or named patient program) provides a mechanism through which physicians can legally and ethically prescribe eteplirsen to patients who meet pre-specified medical criteria and can secure funding. Initially, this limited program was launched in select countries throughout Europe, North America and South America for certain patients where eteplirsen is not currently approved. The Company plans to expand the program to include more countries over time. The program is administered by Clinigen Group plc’s Idis Managed Access division.

As of June 30, 2017, the Company had approximately $301.7 million of cash, cash equivalents and investments, consisting of $168.3 million of cash and cash equivalents, $132.6 million of short-term investments and $0.8 million of restricted cash and investments. The Company believes that, together with the recent equity and debt financings, its balance of cash, cash equivalents and investments as of the date of the issuance of this report is sufficient to fund its current operational plan for at least the next twelve months, though it may pursue additional cash resources through public or private financings, seek additional government funding and establish collaborations with or license its technology to other companies.