Eteplirsen
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| Trade names | Exondys 51 |
| Other names | AVI-4658 |
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| Routes of administration | Intravenous infusion |
| Drug class | Antisense oligonucleotide |
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| Formula | C364H569N177O122P30 |
| Molar mass | 10305.886 g·mol−1 |
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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.[1][2] 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.[3] 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.[4][5] The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) refused to authorize the use of eteplirsen.[6]
- ^ Scoto M, Finkel R, Mercuri E, Muntoni F (August 2018). "Genetic therapies for inherited neuromuscular disorders". The Lancet. Child & Adolescent Health. 2 (8): 600–609. doi:10.1016/S2352-4642(18)30140-8. PMID 30119719. S2CID 52032568.
- ^ Lim KR, Maruyama R, Yokota T (2017). "Eteplirsen in the treatment of Duchenne muscular dystrophy". Drug Design, Development and Therapy. 11: 533–545. doi:10.2147/DDDT.S97635. PMC 5338848. PMID 28280301.
Eteplirsen is applicable for approximately 14% of patients with DMDmutations
- ^ "Perron and UWA to reap $70 million windfall". 3 June 2021. Retrieved 16 November 2021.
- ^ "FDA grants accelerated approval to first drug for Duchenne muscular dystrophy". Press Announcements. U.S. Food & Drug Administration. September 19, 2016. Archived from the original on September 20, 2016. Retrieved September 19, 2016.
- ^ "Railroading at the FDA". Nature Biotechnology. 34 (11): 1078. November 2016. doi:10.1038/nbt.3733. PMID 27824847.
- ^ "Exondys EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 13 August 2020.