eteplirsen

eteplirsen

A proprietary agent in clinical trials for managing Duchenne muscular dystrophy, a condition characterised by defective dystrophin—a key cytoplasmic protein that links muscle fibre cytoskeleton to extracellular matrix through the cell membrane.

Mechanism
Eteplirsen is a splice-switching oligomer that skips exon 51 of the dystrophin gene-DMD, resulting in the production of a shorter, but still functional, dystrophin.
References in periodicals archive ?
A Phase 2 study of Sarepta's drug, eteplirsen, showed a decline in the distance walked for patients at 168 weeks compared with 144 weeks.
In November 2013, the FDA announced that it would not expedite the drug eteplirsen for approval for treatment of Duchenne Muscular Dystrophy while its safety and effectiveness is vetted through more sufficient trial data.
Most surveyed payers have an unfavorable view of confining factors in pivotal clinical trials of emerging Duchenne muscular dystrophy (DMD) treatments eteplirsen (Sarepta Therapeutics) and drisapersen (BioMarin), yet they anticipate generous reimbursement for both agents even at ultra-premium price points.
commercial demand for our lead product candidate eteplirsen in the event of an approval next year, the addition of internal resources will enhance our ability to advance the development of our broader exon skipping platform and explore the potential of our technology platform in other therapeutic areas .
Just this week the US Food and Drugs Administration agreed to consider approval for new drug eteplirsen, which appears to slow the progress of Duchenne.
Orphan disease focus will continue to remain attractive and success of Eteplirsen (PhII, Sarepta) for the treatment of Duchenne muscular dystrophy (DMD) and other antisense, RNAi drugs in the pipeline could see the final maturing of oligonucleotide based drug therapies.
Food and Drug Administration (FDA) has filed the New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, is pleased by today's announcement by Sarepta Therapeutics (formerly AVI BioPharma) that its exon-skipping compound, eteplirsen, achieved significant clinical benefit after 48 weeks of treatment in a Phase IIb study in Duchenne muscular dystrophy.
The anticipated launch of PTC Therapeutics' Translarna in 2015 in the US and 5EU, followed by the 6MM launch of Exon-skipping therapies- BioMarin/Prosensa's drisapersen and Sarepta Therapeutics' eteplirsen, in 2015 and 2016, respectively, are set to change the treatment landscape and drive growth in the DMD market.
It demonstrated for the first time that eteplirsen achieved a highly significant clinical benefit on the 6-minute walk test, over a placebo/delayed treatment cohort in a Phase IIb trial in DMD patients.
AVI BioPharma's eteplirsen, for Duchenne muscular dystrophy: expected release of unblinded data from a Phase IIb trial.
The complaint alleges that the Company made false and/or misleading statements and/or failed to disclose to investors that: (a) the Company failed to provide sufficient data for its New Drug Application submission for marketing approval of eteplirsen, and (b) that as a result, the Application would likely be filed in mid-2015, rather than in 2014.