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Clinigen And Sarepta (SRPT) Launch A Managed Access Program To Treat Patients With Duchenne Muscular Dystrophy Amenable To Exon 51 Skipping

7/19/2017 7:48:15 AM

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BURTON-ON-TRENT, England--(BUSINESS WIRE)--Clinigen Group plc’s (AIM: CLIN, 'Clinigen') Idis Managed Access division and Sarepta Therapeutics Inc. (NASDAQ: SRPT) have initiated a Managed Access Program (MAP) for eteplirsen in certain geographies to treat Duchenne Muscular Dystrophy (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 where funding can be secured.

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 Clinigen Group plc’s Idis Managed Access division. Clinigen is the trusted global leader in access to unlicensed medicines delivering over 220 MAPs to thousands of patients, helping physicians access medicines when no other treatment options are available.

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

For more information on the MAP, including the countries where the program is currently available to patients, please visit


Notes to Editors

About eteplirsen

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 Clinigen Group

Clinigen Group plc (AIM: CLIN) is a global pharmaceutical and services company with a unique combination of businesses focused on providing access to medicines. Its mission is to deliver the right medicine to the right patient at the right time through three areas of global medicine supply; clinical trial, unlicensed and licensed medicines.

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


Clinigen Group plc
Shaun Chilton, Group Chief Executive Officer
Steve Glass, Chief Commercial Officer, North America and Europe
Tel: +44 (0) 1283 495010
Instinctif Partners (media relations - Clinigen)
Melanie Toyne-Sewell / Alex Shaw / Deborah Bell
Tel: +44 (0) 20 7457 2020
Sarepta Therapeutics
Ian Estepan, Media and Investors
Tel: +1 617 274 4052
Brian Reid, W2O Group
Tel: +1 212 257 6725

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