Sarepta Offers Regulatory Update Related to Eteplirsen

Sarepta Therapeutics, Inc.

, a developer of innovative RNA-based therapeutics, today provided an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding its planned New Drug Application (NDA) submission for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). In meeting minutes received last week from a Type B Pre-NDA meeting that took place in September 2014, the FDA provided updated guidance regarding the specific data to be included as part of, or at the time of, Sarepta's NDA submission. The guidance states that additional data are now required as part of the NDA submission, including the results from an independent assessment of dystrophin images and the 168-week clinical data from study 202. Additionally, the guidance requests more specific data including a minimum duration of safety in new patients exposed to eteplirsen, patient-level natural history data to be obtained by Sarepta from independent academic institutions, and MRI data from a recent study conducted by an independent academic group. The FDA indicated that further discussion with Sarepta “will be necessary to determine what would constitute a complete NDA.” Based on these requests, Sarepta plans to submit an NDA by mid-year 2015, pending any additional requests from further discussions with the FDA. "We are committed to satisfying the FDA's updated requests for these specific data to be included as part of an NDA submission and will continue to work with the Agency toward the goal of a complete and acceptable NDA filing," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "We believe all of the data requests and additional FDA discussions that have currently been outlined can be completed in time for an NDA submission by mid-year 2015. Obtaining an FDA approval of eteplirsen for the DMD patients who may benefit from the drug continues to be our highest priority.” Excerpts from the Pre-NDA Meeting Minutes related to information that the FDA is requesting as part of an NDA submission included: "The sponsor should include 3-month data from at least 12 to 24 newly exposed patients at the time the NDA is submitted." "Available data from the other patients enrolled in the new eteplirsen studies (studies 301, 203, 204) should also be included at the time the NDA is submitted, even if exposure is less than 3 months in duration." "Additional data from later time points and from newly enrolled patients should be submitted in the 120-Day Safety Update." "FDA strongly advises the sponsor to obtain and submit patient-level natural history data. FDA is prepared to appeal to the academic groups holding the data to allow the sponsor a means to acquire the data." "The study 201/202 clinical site inspection conducted in May, 2014, after the issuance of the April 15, 2014, guidance letter, uncovered marked disparities in the immunohistochemistry methodology and concerns about the reproducibility of the data. The lack of confirmation of robust dystrophin measurement during the site visit necessitates including the independent assessment of dystrophin-positive fibers and 168-week efficacy data from study 201/202 in the NDA." “FDA strongly urged the sponsor to submit the MRI data with appropriate natural history controls.” The FDA also stated that “[a]dditional discussion between the sponsor and the FDA will be necessary to determine what would constitute a complete NDA.” Conference Call Information Sarepta will hold a conference call to discuss this update today at 8:00 a.m. EDT (5:00 a.m. PDT). The conference call may be accessed by dialing 800.708.4539 for domestic callers and 847.619.6396 for international callers. The passcode for the call is 38376370. Please specify to the operator that you would like to join the "Sarepta Regulatory Update Call." The conference call will be webcast live under the investor relations section of Sarepta's website at www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. An audio replay will be available through November 3, 2014 by calling 888.843.7419 or 630.652.3042 and entering access code 38376370.