eteplirsen
Sign in to saveAlso known as AVI-4658, Exondys 51®, AVI 4658
Eteplirsen (brand name Exondys 51) is a medication to treat, but not cure, some types of Duchenne muscular dystrophy (DMD), caused by a specific mutation. Eteplirsen only targets specific mutations and can be used to treat about 14% of DMD cases. Eteplirsen is a form of antisense therapy.
Wikidata facts
- Subclass of
- chemical compound
- Has use
- medication
Show 5 more facts
- chemical formula
- C₃₆₄H₅₆₉N₁₇₇O₁₂₂P₃₀
- World Health Organisation international non-proprietary name
- eteplirsen
- medical condition treated
- Duchenne muscular dystrophy
- route of administration
- intravenous infusion and defusion
- subject has role
- small interfering RNA
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Encyclopedic overview
9 sectionsContents
- Adverse effects
- Mechanism of action
- Nature and sequence of oligo and target
- Pharmacokinetics
- History
- Society and culture
- Economics
- References
- External links
Eteplirsen (brand name Exondys 51) is a medication to treat, but not cure, some types of Duchenne muscular dystrophy (DMD), caused by a specific mutation. Eteplirsen only targets specific mutations and can be used to treat about 14% of DMD cases. Eteplirsen is a form of antisense therapy.
Eteplirsen was developed by Steve Wilton, Sue Fletcher and colleagues at the University of Western Australia and commercialized by Sarepta Therapeutics. After a controversial debate surrounding the drug's efficacy, during which two FDA review panel members resigned in protest, eteplirsen received accelerated approval from the US Food and Drug administration in late 2016. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) refused to authorize the use of eteplirsen.
Excerpted from Wikipedia’s “eteplirsen” article, available under the CC BY-SA 4.0 licence.