Eteplirsen

Eteplirsen
Clinical data
Trade namesExondys 51
Other namesAVI-4658
License data
Routes of
administration		Intravenous infusion
Drug classAntisense oligonucleotide
ATC code
Legal status
Legal status
Identifiers
  • (P-deoxy-P-(dimethylamino))(2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G),5'-(P-(4-((2-(2-(2-hydroxyethoxy)ethoxy)ethoxy)carbonyl)-1-piperazinyl)-N,N-dimethylphosphonamidate) RNA
CAS Number
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
Chemical and physical data
FormulaC364H569N177O122P30
Molar mass10305.886 g·mol−1
  • Cc1cn(c(=O)[nH]c1=O)[C@H]2CN(C[C@H](O2)COP(=O)(N3C[C@H](O[C@H](C3)n4ccc(nc4=O)N)COP(=O)(N5CCN(CC5)C(=O)OCCOCCOCCO)N(C)C)N(C)C)P(=O)(N(C)C)OC[C@@H]6CN(C[C@@H](O6)n7ccc(nc7=O)N)P(=O)(N(C)C)OC[C@@H]8CN(C[C@@H](O8)n9ccc(nc9=O)N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1ncnc2N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1ncnc2N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1ccc(nc1=O)N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1ncnc2N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cc(c(=O)[nH]c1=O)C)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1ccc(nc1=O)N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1ncnc2N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1ncnc2N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1nc([nH]c2=O)N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1nc([nH]c2=O)N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1ncnc2N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1ncnc2N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1nc([nH]c2=O)N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1ncnc2N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cc(c(=O)[nH]c1=O)C)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1nc([nH]c2=O)N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1nc([nH]c2=O)N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1ccc(nc1=O)N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1ncnc2N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cc(c(=O)[nH]c1=O)C)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cc(c(=O)[nH]c1=O)C)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cc(c(=O)[nH]c1=O)C)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1ccc(nc1=O)N)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cc(c(=O)[nH]c1=O)C)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1cnc2c1ncnc2N)P(=O)(N(C)C)OC[C@@H]1CNC[C@@H](O1)n1cnc2c1nc([nH]c2=O)N
  • InChI=1S/C364H569N177O122P30/c1-215-94-519(357(563)444-331(215)543)267-137-490(108-230(642-267)167-612-667(574,454(14)15)483-101-223(635-260(130-483)512-75-68-252(365)425-350(512)556)160-605-664(571,451(8)9)482-84-82-481(83-85-482)364(570)603-91-90-602-89-88-601-87-86-542)673(580,460(26)27)607-162-225-103-484(131-261(637-225)513-76-69-253(366)426-351(513)557)666(573,453(12)13)606-161-224-102-486(133-263(636-224)515-78-71-255(368)428-353(515)559)669(576,456(18)19)621-176-240-118-502(149-279(652-240)532-204-414-294-310(377)394-194-404-320(294)532)684(591,471(48)49)625-179-242-120-498(145-275(654-242)528-200-410-290-306(373)390-190-400-316(290)528)680(587,467(40)41)610-165-228-106-488(135-265(640-228)517-80-73-257(370)430-355(517)561)671(578,458(22)23)619-174-238-116-499(146-276(650-238)529-201-411-291-307(374)391-191-401-317(291)529)681(588,468(42)43)616-171-234-112-492(139-269(646-234)521-96-217(3)333(545)446-359(521)565)675(582,462(30)31)609-164-227-105-487(134-264(639-227)516-79-72-256(369)429-354(516)560)670(577,457(20)21)622-177-241-119-503(150-280(653-241)533-205-415-295-311(378)395-195-405-321(295)533)685(592,472(50)51)626-180-243-122-506(153-282(655-243)535-207-417-297-313(380)397-197-407-323(297)535)688(595,475(56)57)631-186-249-128-511(158-288(661-249)541-214-424-304-330(541)437-349(387)443-343(304)555)693(600,480(66)67)633-188-251-129-509(156-287(663-251)540-213-423-303-329(540)436-348(386)442-342(303)554)691(598,478(62)63)629-183-246-124-504(151-281(658-246)534-206-416-296-312(379)396-196-406-322(296)534)686(593,473(52)53)627-181-244-123-505(152-283(656-244)536-208-418-298-314(381)398-198-408-324(298)536)687(594,474(54)55)630-185-248-127-508(155-286(660-248)539-212-422-302-328(539)435-347(385)441-341(302)553)690(597,477(60)61)628-182-245-121-501(148-278(657-245)531-203-413-293-309(376)393-193-403-319(293)531)683(590,470(46)47)618-173-236-114-496(143-273(648-236)525-100-221(7)337(549)450-363(525)569)679(586,466(38)39)624-184-247-125-510(157-285(659-247)538-211-421-301-327(538)434-346(384)440-340(301)552)692(599,479(64)65)632-187-250-126-507(154-284(662-250)537-210-420-300-326(537)433-345(383)439-339(300)551)689(596,476(58)59)611-166-229-107-489(136-266(641-229)518-81-74-258(371)431-356(518)562)672(579,459(24)25)620-175-239-117-500(147-277(651-239)530-202-412-292-308(375)392-192-402-318(292)530)682(589,469(44)45)617-172-235-113-494(141-271(647-235)523-98-219(5)335(547)448-361(523)567)677(584,464(34)35)615-170-233-111-493(140-270(645-233)522-97-218(4)334(546)447-360(522)566)676(583,463(32)33)614-169-232-110-491(138-268(644-232)520-95-216(2)332(544)445-358(520)564)674(581,461(28)29)608-163-226-104-485(132-262(638-226)514-77-70-254(367)427-352(514)558)668(575,455(16)17)613-168-231-109-495(142-272(643-231)524-99-220(6)336(548)449-362(524)568)678(585,465(36)37)623-178-237-115-497(144-274(649-237)527-199-409-289-305(372)389-189-399-315(289)527)665(572,452(10)11)604-159-222-92-388-93-259(634-222)526-209-419-299-325(526)432-344(382)438-338(299)550/h68-81,94-100,189-214,222-251,259-288,388,542H,82-93,101-188H2,1-67H3,(H2,365,425,556)(H2,366,426,557)(H2,367,427,558)(H2,368,428,559)(H2,369,429,560)(H2,370,430,561)(H2,371,431,562)(H2,372,389,399)(H2,373,390,400)(H2,374,391,401)(H2,375,392,402)(H2,376,393,403)(H2,377,394,404)(H2,378,395,405)(H2,379,396,406)(H2,380,397,407)(H2,381,398,408)(H,444,543,563)(H,445,544,564)(H,446,545,565)(H,447,546,566)(H,448,547,567)(H,449,548,568)(H,450,549,569)(H3,382,432,438,550)(H3,383,433,439,551)(H3,384,434,440,552)(H3,385,435,441,553)(H3,386,436,442,554)(H3,387,437,443,555)/t222-,223-,224-,225-,226-,227-,228-,229-,230-,231-,232-,233-,234-,235-,236-,237-,238-,239-,240-,241-,242-,243-,244-,245-,246-,247-,248-,249-,250-,251-,259+,260+,261+,262+,263+,264+,265+,266+,267+,268+,269+,270+,271+,272+,273+,274+,275+,276+,277+,278+,279+,280+,281+,282+,283+,284+,285+,286+,287+,288+,664?,665?,666?,667?,668?,669?,670?,671?,672?,673?,674?,675?,676?,677?,678?,679?,680?,681?,682?,683?,684?,685?,686?,687?,688?,689?

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]

  1. ^ 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.
  2. ^ 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
  3. ^ "Perron and UWA to reap $70 million windfall". 3 June 2021. Retrieved 16 November 2021.
  4. ^ "FDA grants accelerated approval to first drug for Duchenne muscular dystrophy". Press Announcements. U.S. Food & Drug Administration. September 19, 2016. Retrieved September 19, 2016.
  5. ^ "Railroading at the FDA". Nature Biotechnology. 34 (11): 1078. November 2016. doi:10.1038/nbt.3733. PMID 27824847.
  6. ^ "Exondys EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 13 August 2020.