Sarepta Therapeutics and Clinigen Launch a Managed Access Program to Treat Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
07/19/17 8:30 AM EDT
Initially, the program is being launched in select countries within Europe, North America and South America for certain patients where eteplirsen is not currently approved. Sarepta plans to expand the program to include more countries over time.
The program will be administered by
All requests must be submitted by the treating physician on behalf of the patient. Healthcare providers can obtain details about the EXONDYS 51® (eteplirsen) Managed Access Program by calling a Clinigen representative at +44 1283 494 340, or emailing firstname.lastname@example.org.
For more information on the MAP, including the countries where the program is currently available to patients, please visit www.sarepta.com/community/managed-access-program.
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 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 – 5,000 males 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 of the body. The condition is universally fatal, and death usually occurs before the age of 30 generally due to respiratory or cardiac failure.
About Sarepta Therapeutics
Sarepta Therapeutics is a U.S. 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 Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit www.sarepta.com.
About Clinigen Group
This press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements about the MAP being a mechanism through which physicians can legally and ethically prescribe eteplirsen to eligible patients who meet pre-specified medical criteria and where funding can be secured; and Sarepta’s plan to expand the Managed Access Program it initiated with Clinigen to include more countries over time.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others: Sarepta, in partnership with Clinigen, may not be able to establish and successfully conduct a Managed Access Program in one or more countries, and even if such program(s) are successfully conducted in each country targeted, Sarepta may not achieve any significant revenues from sales of eteplirsen under the MAP in one or more of the countries in which the MAP is launched; and Sarepta may not be able to commercialize eteplirsen in MAP countries even if the product has been available on a named patient basis. Any of the foregoing risks could 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 Sarepta's 2016 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q for the quarter ended
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Media and Investors:
Sarepta Therapeutics, Inc.Ian Estepan, 617-274-4052 email@example.com or W2O GroupBrian Reid, 212-257-6725 firstname.lastname@example.org or Clinigen Group plcShaun Chilton / Martin Abell, +44 1283 495 010 or Instinctif Partners(Clinigen Media Relations) Melanie Toyne-Sewell / Alex Shaw / Deborah Bell, +44 2074 572 020 email@example.com
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