eteplirsen (Exondys)
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Indications
- Duchenne muscular dystrophy (accelerated FDA approval)
Dosage
- intravenous infusion 50 mg/mL (0.5-1 mL)
Pharmacokinetics
- not metabolized
- renal elimination
- protein binding < 16%
Mechanism of action
- targets mutations in a region implicated in 13% of Duchenne muscular dystrophy cases
- morpholino antisense oligomer triggering excision of exon 51 during pre-mRNA splicing of the dystrophin RNA transcript
- this changes the downstream reading frame of dystrophin
- eteplirsen given to a healthy person would result in a non-functional dystrophin protein
- for Duchenne muscular dystrophy patients with particular frameshifting mutations, eteplirsen can restore the mRNA reading frame resulting in a functional (albiet modified) dystrophin
More general terms
References
- ↑ Wikipedia: Eteplirsen https://en.wikipedia.org/wiki/Eteplirsen
- ↑ Cirak S, Arechavala-Gomeza V, Guglieri M et al Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose- escalation study. Lancet. 2011 Aug 13;378(9791):595-605. Epub 2011 Jul 23. PMID: https://www.ncbi.nlm.nih.gov/pubmed/21784508 Free PMC Article
- ↑ FDA News Release. September 19, 2016 FDA grants accelerated approval to first drug for Duchenne muscular dystrophy. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm521263.htm
- ↑ Fiore K Eteplirsen Boosts Dystrophin, Function in DMD - Details from 12 patients in Study 201/202 reported at AAN. MedPage Today. April 27, 2017 https://www.medpagetoday.com/MeetingCoverage/AAN/64874
Mendell JR, et al Exon skipping: Development and approval of eteplirsen for DMD American Academy of Neurology (AAN) Annual Meeting 2017