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Substance Name: Eteplirsen [USAN:INN]
RN: 1173755-55-9
UNII: AIW6036FAS
InChIKey: KZOVCSBYYXUVDW-UHFFFAOYSA-N

Notes

  • A phosphorodiamidate morpholino oligomer used in the treatment of DUCHENNE MUSCULAR DYSTROPHY with genetic mutations amenable to skipping exon 51.
  • Treatment of Duchenne muscular dystrophy.

Molecular Formula

  • C364-H569-N177-O122-P30

Molecular Weight

  • 10369.8321
 

Classification Code

  • Treatment of Duchenne Muscular Dystrophy
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Names and Synonyms

Name of Substance

  • Eteplirsen [USAN:INN]

Synonyms

  • AVI 4658
  • AVI-4658
  • Eteplirsen
  • Exondys 51
  • UNII-AIW6036FAS

Systematic Names

  • RNA, (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, (P-deoxy-P-(dimethylamino))(2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a5')(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)

Registry Numbers

CAS Registry Number

  • 1173755-55-9

FDA UNII

  • AIW6036FAS

System Generated Number

  • 1173755559

Structure Descriptors

InChI

1S/C364H569N177O126P30/c1-215-94-519(357(563)444-331(215)543)267-137-483(670(573,453(12)13)610-165-228-106-488(131-261(640-228)513-76-69-253(366)426-351(513)557)675(578,458(22)23)616-171-234-112-495(141-271(647-234)523-98-219(5)335(547)448-361(523)567)682(585,465(36)37)624-177-237-115-497(146-276(651-237)529-201-411-291-307(374)391-191-401-317(291)529)669(572,452(10)11)608-163-222-92-388-93-259(634-222)526-209-419-299-325(526)432-344(382)438-338(299)550)101-223(635-267)159-604-665-695(598,478(62)63)509-127-249(650-273(143-509)525-100-221(7)337(549)450-363(525)569)186-631-683(586,466(38)39)496-114-236(649-272(142-496)524-99-220(6)336(548)449-362(524)568)173-618-686(589,469(44)45)500-118-240(654-279(149-500)532-204-414-294-310(377)394-194-404-320(294)532)176-623-677(580,460(26)27)490-110-232(644-263(133-490)515-78-71-255(368)428-353(515)559)169-614-689(592,472(50)51)503-123-246(662-284(152-503)537-210-420-300-326(537)433-345(383)439-339(300)551)183-628-691(594,474(54)55)505-121-243(659-285(154-505)538-211-421-301-327(538)434-346(384)440-340(301)552)180-619-671(574,454(14)15)484-102-224(636-268(138-484)520-95-216(2)332(544)445-358(520)564)160-605-666-696(599,479(64)65)510-129-251(658-283(157-510)536-208-418-298-314(381)398-198-408-324(298)536)188-633-693(596,476(58)59)507-125-245(661-288(156-507)541-214-424-304-330(541)437-349(387)443-343(304)555)182-627-688(591,471(48)49)502-119-241(655-281(151-502)534-206-416-296-312(379)396-196-406-322(296)534)178-625-687(590,470(46)47)501-120-242(656-280(150-501)533-205-415-295-311(378)395-195-405-321(295)533)179-626-690(593,473(52)53)504-124-247(663-287(153-504)540-213-423-303-329(540)436-348(386)442-342(303)554)184-629-692(595,475(56)57)506-122-244(660-286(155-506)539-212-422-302-328(539)435-347(385)441-341(302)553)181-621-673(576,456(18)19)486-104-226(638-275(145-486)528-200-410-290-306(373)390-190-400-316(290)528)162-607-667-697(600,480(66)67)511-128-250(657-282(158-511)535-207-417-297-313(380)397-197-407-323(297)535)187-632-679(582,462(30)31)492-108-230(642-265(135-492)517-80-73-257(370)430-355(517)561)167-612-681(584,464(34)35)494-113-235(648-270(140-494)522-97-218(4)334(546)447-360(522)566)172-617-685(588,468(42)43)499-117-239(653-278(148-499)531-203-413-293-309(376)393-193-403-319(293)531)175-622-676(579,459(24)25)489-109-231(643-262(132-489)514-77-70-254(367)427-352(514)558)168-613-684(587,467(40)41)498-116-238(652-277(147-498)530-202-412-292-308(375)392-192-402-318(292)530)174-620-672(575,455(16)17)485-103-225(637-274(144-485)527-199-409-289-305(372)389-189-399-315(289)527)161-606-664-694(597,477(60)61)508-126-248(645-266(136-508)518-81-74-258(371)431-356(518)562)185-630-678(581,461(28)29)491-107-229(641-264(134-491)516-79-72-256(369)429-354(516)560)166-611-680(583,463(32)33)493-111-233(646-269(139-493)521-96-217(3)333(545)446-359(521)565)170-615-674(577,457(20)21)487-105-227(639-260(130-487)512-75-68-252(365)425-350(512)556)164-609-668(571,451(8)9)482-84-82-481(83-85-482)364(570)603-91-90-602-89-88-601-87-86-542/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)

InChIKey

KZOVCSBYYXUVDW-UHFFFAOYSA-N