Clinigen & Sarepta Therapeutics launch a Managed Access Program to treat patients with Duchenne Muscular Dystrophy amenable t...
July 19 2017 - 8:41AM
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
medicine.access@clinigengroup.com.
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.
-Ends-
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 www.sarepta.com.
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Clinigen Group plcShaun Chilton, Group Chief Executive
OfficerorSteve Glass, Chief Commercial Officer, North America and
EuropeTel: +44 (0) 1283 495010orInstinctif Partners (media
relations - Clinigen)Melanie Toyne-Sewell / Alex Shaw / Deborah
BellTel: +44 (0) 20 7457
2020Email:clinigen@instinctif.comorSarepta TherapeuticsIan
Estepan, Media and InvestorsTel: +1 617 274 4052Email:
iestepan@sarepta.comorBrian Reid, W2O GroupTel: +1 212 257
6725Email: breid@w2ogroup.com
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