Eteplirsen: First Global Approval
Eteplirsen (Exondys 51) is an antisense oligonucleotide designed to induce exon 51 skipping that is developed by Sarepta Therapeutics. Intravenous eteplirsen has received accelerated approval from the US FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation...
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| Veröffentlicht in: | Drugs (New York, N.Y.) N.Y.), 2016-11, Vol.76 (17), p.1699-1704 |
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| creator | Syed, Yahiya Y. |
| description | Eteplirsen (Exondys 51) is an antisense oligonucleotide designed to induce exon 51 skipping that is developed by Sarepta Therapeutics. Intravenous eteplirsen has received accelerated approval from the US FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the
DMD
gene amenable to exon 51 skipping. Eteplirsen has orphan drug designation in the USA and EU, and rare paediatric disease designation in the USA for use in DMD. In the phase III PROMOVI trial, eteplirsen significantly increased dystrophin levels from baseline in muscle tissues of 12 evaluable patients with DMD after 48 weeks of treatment. This finding is supported by data from phase II trials. Long-term treatment with eteplirsen was associated with a decrease in the rate of decline in ambulation and pulmonary function in an open-label extension of a phase II trial. Eteplirsen was generally well tolerated in clinical trials. This article summarizes the milestones in the development of eteplirsen leading to this first approval for DMD. |
| doi_str_mv | 10.1007/s40265-016-0657-1 |
| format | Article |
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DMD
gene amenable to exon 51 skipping. Eteplirsen has orphan drug designation in the USA and EU, and rare paediatric disease designation in the USA for use in DMD. In the phase III PROMOVI trial, eteplirsen significantly increased dystrophin levels from baseline in muscle tissues of 12 evaluable patients with DMD after 48 weeks of treatment. This finding is supported by data from phase II trials. Long-term treatment with eteplirsen was associated with a decrease in the rate of decline in ambulation and pulmonary function in an open-label extension of a phase II trial. Eteplirsen was generally well tolerated in clinical trials. This article summarizes the milestones in the development of eteplirsen leading to this first approval for DMD.</description><identifier>ISSN: 0012-6667</identifier><identifier>EISSN: 1179-1950</identifier><identifier>DOI: 10.1007/s40265-016-0657-1</identifier><identifier>PMID: 27807823</identifier><language>eng</language><publisher>Cham: Springer International Publishing</publisher><subject>AdisInsight Report ; Agreements ; Biomarkers ; Clinical trials ; Clinical Trials, Phase II as Topic ; Clinical Trials, Phase III as Topic ; Drug Approval ; Drug dosages ; Humans ; Internal Medicine ; Medicine ; Medicine & Public Health ; Morpholinos - therapeutic use ; Muscular dystrophy ; Muscular Dystrophy, Duchenne - drug therapy ; Mutation ; Oligonucleotides, Antisense - therapeutic use ; Pediatrics ; Pharmacokinetics ; Pharmacology/Toxicology ; Pharmacotherapy ; United States ; United States Food and Drug Administration</subject><ispartof>Drugs (New York, N.Y.), 2016-11, Vol.76 (17), p.1699-1704</ispartof><rights>Springer International Publishing Switzerland 2016</rights><rights>Copyright Springer Science & Business Media Nov 2016</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c405t-a4333d3fe2b9feb72d58fa086ce94a7fce8964c18fa43680b614cd2ac8d607b33</citedby><cites>FETCH-LOGICAL-c405t-a4333d3fe2b9feb72d58fa086ce94a7fce8964c18fa43680b614cd2ac8d607b33</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1007/s40265-016-0657-1$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s40265-016-0657-1$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,780,784,27922,27923,41486,42555,51317</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/27807823$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Syed, Yahiya Y.</creatorcontrib><title>Eteplirsen: First Global Approval</title><title>Drugs (New York, N.Y.)</title><addtitle>Drugs</addtitle><addtitle>Drugs</addtitle><description>Eteplirsen (Exondys 51) is an antisense oligonucleotide designed to induce exon 51 skipping that is developed by Sarepta Therapeutics. Intravenous eteplirsen has received accelerated approval from the US FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the
DMD
gene amenable to exon 51 skipping. Eteplirsen has orphan drug designation in the USA and EU, and rare paediatric disease designation in the USA for use in DMD. In the phase III PROMOVI trial, eteplirsen significantly increased dystrophin levels from baseline in muscle tissues of 12 evaluable patients with DMD after 48 weeks of treatment. This finding is supported by data from phase II trials. Long-term treatment with eteplirsen was associated with a decrease in the rate of decline in ambulation and pulmonary function in an open-label extension of a phase II trial. Eteplirsen was generally well tolerated in clinical trials. 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Academic</collection><jtitle>Drugs (New York, N.Y.)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Syed, Yahiya Y.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Eteplirsen: First Global Approval</atitle><jtitle>Drugs (New York, N.Y.)</jtitle><stitle>Drugs</stitle><addtitle>Drugs</addtitle><date>2016-11-01</date><risdate>2016</risdate><volume>76</volume><issue>17</issue><spage>1699</spage><epage>1704</epage><pages>1699-1704</pages><issn>0012-6667</issn><eissn>1179-1950</eissn><abstract>Eteplirsen (Exondys 51) is an antisense oligonucleotide designed to induce exon 51 skipping that is developed by Sarepta Therapeutics. Intravenous eteplirsen has received accelerated approval from the US FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the
DMD
gene amenable to exon 51 skipping. Eteplirsen has orphan drug designation in the USA and EU, and rare paediatric disease designation in the USA for use in DMD. In the phase III PROMOVI trial, eteplirsen significantly increased dystrophin levels from baseline in muscle tissues of 12 evaluable patients with DMD after 48 weeks of treatment. This finding is supported by data from phase II trials. Long-term treatment with eteplirsen was associated with a decrease in the rate of decline in ambulation and pulmonary function in an open-label extension of a phase II trial. Eteplirsen was generally well tolerated in clinical trials. This article summarizes the milestones in the development of eteplirsen leading to this first approval for DMD.</abstract><cop>Cham</cop><pub>Springer International Publishing</pub><pmid>27807823</pmid><doi>10.1007/s40265-016-0657-1</doi><tpages>6</tpages></addata></record> |
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| subjects | AdisInsight Report Agreements Biomarkers Clinical trials Clinical Trials, Phase II as Topic Clinical Trials, Phase III as Topic Drug Approval Drug dosages Humans Internal Medicine Medicine Medicine & Public Health Morpholinos - therapeutic use Muscular dystrophy Muscular Dystrophy, Duchenne - drug therapy Mutation Oligonucleotides, Antisense - therapeutic use Pediatrics Pharmacokinetics Pharmacology/Toxicology Pharmacotherapy United States United States Food and Drug Administration |
| title | Eteplirsen: First Global Approval |
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