Negative opinion for EXONDYS® in Europe


Here’s the latest news from Sarepta Therapeutics press release about EXONDYS® (eteplirsen). EXONDYS® (eteplirsen) is designed to treat patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51 of the dystrophin gene. Enjoy reading and thank you for sharing these articles within the DMD community.


Sarepta receives negative CHMP opinion for EXONDYS® (eteplirsen) in Europe

Sarepta Therapeutics, Inc., announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had adopted a negative opinion for EXONDYS® (eteplirsen).


Request for re-examination

Sarepta will request a re-examination of the opinion, which will result in the assignment of a new rapporteur and co-rapporteur. The company will also request a Scientific Advisory Group (SAG) on DMD to be called so that neuromuscular specialists experienced with working on treatments for these patients can provide expert guidance and insight into, among other things, the validity of the external controls used and the importance of certain functional endpoints, including, for instance, the relevance of meaningful slowing pulmonary decline in patients with this difficult to treat disease. The re-examination process is expected to be completed by year-end 2018.


EXONDYS® a treatment using the exon-skipping technique

This treatment uses a specific exon-skipping technique to jump over a portion of genetic machinery that produces a non-working, mutated form of dystrophin in children with DMD. It aims to restore the machinery’s ability to read genetic code, so it can produce a less mutated form of dystrophin that works in children with DMD.

The production of partly functional dystrophin may delay muscle destruction and extend mobility in children with this devastating, rare disease. More specifically, Exondys 51 (eteplirsen) triggers the skipping of exon 51, which occurs in 13% of children with DMD.


About DMD

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.


More interesting link:

Please visit: www.sarepta.com

Video Professor Georges Dickson

Press Release

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