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 ?
EXONDYS 51(eteplirsen) continues to perform, with second quarter sales at $94.7 million, 29% growth quarter over same quarter last year.
EXONDYS 51(R)(eteplirsen) continues to perform, with second quarter sales at $94.7 million, 29% growth quarter over same quarter last year.
PTC's Ataluren and Accelerated Approval: Is Eteplirsen A Precedent After All?
Antisense oligonucleotides are a third noteworthy category of products; not least because newly approved eteplirsen and nusinersen fall into this segment.
Or of Sarepta's eteplirsen drug product for Duchenne muscular dystrophy, which was embroiled in controversy within FDA for its approval, for which many members internally sought to reject its approval initially.
There are also novel therapies like gene therapy, eteplirsen (an antisense oligonucleotide), ataluren (an investigational orally administered drug), creatinine, myostatin inactivation, cell therapy, and idebenone, but none of them have yet provided a definitive treatment of DMD [14-20].
The first quarter of 2017 showed significant progress across all areas of the business, including executing a strong launch for EXONDYS 51 (eteplirsen), building our pipeline, working to expand globally, and forging strategic partnerships that support our goal to help as many DMD patients as possible, said Edward Kaye, Sareptas chief executive officer.
Eteplirsen (Exondys 51) is an antisense oligonucleotide that is given intravenously.
Allowed staff to approve eteplirsen to treat muscular dystrophy, despite evidence that it didn't produce clinical improvement (2016, President Obama, Commissioner Califf).
Recently, the FDA conditionally approved Eteplirsen (Exondy51) [26], an AON that skips exon 51, but its half-life in blood is only 3 hours, hence a weekly injection is required.