|Sarepta Therapeutics Announces FDA Will Consider Accelerated Approval for Eteplirsen After Further Review of Data on Dystrophin and Clinical Outcomes|
Specifically, Sarepta received feedback on both the acceptability of dystrophin as a surrogate endpoint that would reasonably predict clinical benefit in DMD patients and the acceptability of the eteplirsen safety database for a Subpart H Accelerated Approval filing.
"We are encouraged by our interactions with the
At the End-of-Phase II Clinical Meeting in March, there was a productive discussion on the suitability of dystrophin as a surrogate marker, including a presentation by Sarepta detailing the methodologies used to analyze dystrophin in the studies and supportive data suggesting that the dystrophin produced is functional. As follow up to the discussion, and as reflected in the meeting minutes from the
Furthermore, the meeting minutes contained the following statement:
"The Agency stated that they had not made a final decision regarding acceptability of the proposed Subpart H (Accelerated Approval) NDA filing, and that the Agency would consider the additional data submitted by the sponsor before making a final decision."
Sarepta also discussed the eteplirsen and phosphorodiamidate morpholino oligomer (PMO) safety database at the End-of-Phase II meeting and asked if the 38 patient eteplirsen safety database was sufficient for a Subpart H Accelerated Approval filing. While Sarepta will continue to collect long-term safety from the ongoing eteplirsen extension study for a potential submission, the meeting minutes stated that: "In the event (the Agency) agrees to file the Subpart H NDA submission, additional safety data to support approval could come from the first few months of the... pivotal confirmatory study." Sarepta still intends to begin dosing patients in a pivotal confirmatory study in the first quarter of 2014.
Sarepta is preparing to submit the dystrophin and clinical outcomes summaries and will be requesting a follow-up meeting with the
Conference Call Information
Sarepta will hold a conference call to discuss this update today at
About Duchenne Muscular Dystrophy
DMD is an X-linked rare, degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 boys worldwide. A devastating and incurable muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Progressive muscle weakness in the lower limbs eventually spreads to the arms, neck and other areas. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and death usually occurs before the age of 30.
Eteplirsen is Sarepta's lead drug candidate that is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Eteplirsen uses Sarepta's phosphorodiamidate morpholino oligomer (PMO)-based chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13 percent of the total DMD population. By skipping exon 51, eteplirsen may restore the gene's ability to make a shorter, but still functional, form of dystrophin from messenger RNA, or mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to improve, stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD.
About the Accelerated Approval Regulatory Pathway (21 CFR 314 Subpart H)
To speed the development and availability of new medicines for serious and life-threatening diseases, the
Forward Looking Statement
This press release contains forward-looking statements. These forward-looking statements generally can be identified by use of words such as "believes or belief," "anticipates," "plans," "expects," "will," "intends," "potential," "possible," "advance" and similar expressions. These forward-looking statements include statements about the development of eteplirsen and its efficacy, safety, potency and utility in the treatment of DMD; the potential for the creation of novel dystrophin to lead to significant clinical benefit or its suitability as a valid surrogate marker; the amount and type of dystrophin, safety and other information necessary for the Agency to make regulatory determinations; the impact of manufacturing and development activities on NDA submission timelines; and the potential use of the accelerated approval pathway for eteplirsen.
Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: subsequent clinical trials may fail to demonstrate the safety and efficacy of eteplirsen or replicate results; treatment of patients with DMD using eteplirsen over a longer duration may not lead to significant clinical benefit; any of Sarepta's drug candidates, including eteplirsen, may fail in development, may not qualify for filing under Subpart H accelerated approval or receive required regulatory approvals at all, or may not become commercially viable due to delays, decisions by regulatory authorities, or other reasons; and those identified under the heading "Risk Factors" in Sarepta's Annual Report on Form 10-K for the full year ended
Any of the foregoing risks could materially and adversely affect Sarepta's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the Company's filings with the
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