Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious, and other diseases.
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|Sarepta Therapeutics Announces Regulatory Update on Eteplirsen|
Updated and additional guidance received from
NDA submission planned for mid-year 2015;
Company to hold teleconference today at
In meeting minutes received last week from a Type B Pre-NDA meeting that
took place in
"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
Excerpts from the Pre-NDA Meeting Minutes related to information that
"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."
"The study 201/202 clinical site inspection conducted in May, 2014,
after the issuance of the
“FDA strongly urged the sponsor to submit the MRI data with appropriate natural history controls.”
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. DMD affects approximately one in every 3,500 boys born 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 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 and is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.
Eteplirsen uses Sarepta's novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip mutations affecting exon 51 of the dystrophin gene. Approximately 13 percent of the total DMD population is amenable to exon 51 skipping. 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 stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD. Sarepta is also developing other PMO-based exon-skipping drug candidates intended to treat additional patients with DMD.
Forward-Looking Statements and Information
This press release contains forward-looking statements. These
forward-looking statements generally can be identified by the 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
Sarepta’s planned timing for an NDA submission for eteplirsen in the
treatment of DMD; Sarepta’s plans to work with the
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: we
may not be able to comply with all
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
“Safe Harbor” Statement under the Private Securities Litigation Reform Act of 1995: The statements that are not historical facts contained in this release are forward–looking statements that involve risks and uncertainties, including, but not limited to, the results of research and development efforts, the results of preclinical and clinical testing, the effect of regulation by the FDA and other agencies, the impact of competitive products, product development, commercialization and technological difficulties, and other risks detailed in the company’s Securities and Exchange Commission filings.