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 ?
The Phase 1 inclusion criteria allow for participation of both ambulatory and non-ambulatory patients, including those previously treated with eteplirsen following an appropriate washout period.
Eteplirsen (Exondys 51) is an antisense oligonucleotide that is given intravenously.
RNA therapeutics company Sarepta Therapeutics (NasdaqGS:SRPT) reported on Monday the receipt of the European Medicines Agency's (EMA) validation for its previous Marketing Authorization application (MAA) for eteplirsen for treatment of Duchenne muscular dystrophy amenable to exon 51 skipping.
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.
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.
However, it remains to be seen whether eteplirsen can maintain a significant clinical benefit with time.
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.
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.
United States-based Sarepta Therapeutics has dosed the first patient in a clinical trial of its lead exon-skipping therapeutic candidate, eteplirsen, to treat duchenne muscular dystrophy, it was reported yesterday.
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 .
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.