Eteplirsen

Eteplirsen
Clinical data
Trade names	Exondys 51
Other names	AVI-4658
License data	US DailyMed: Eteplirsen;
Routes of; administration	Intravenous infusion
Drug class	Antisense oligonucleotide
ATC code	M09AX06 (WHO) ;
Legal status
Legal status	US: ℞-only;
Identifiers
	IUPAC name (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	1173755-55-9;
DrugBank	DB06014;
ChemSpider	34983391;
UNII	AIW6036FAS;
KEGG	D09900;
ChEMBL	ChEMBL2108278;
Chemical and physical data
Formula	C364H569N177O122P30
Molar mass	10305.886 g·mol−1
	SMILES 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 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. 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.

This article is issued from Wikipedia. The text is licensed under Creative Commons - Attribution - Sharealike. Additional terms may apply for the media files.