Volanesorsen

Volanesorsen
Clinical data
Trade namesWaylivra
Other namesISIS 304801, ISIS-APOCIIIRx
Routes of
administration		Subcutaneous
ATC code
Legal status
Legal status
  • UK: POM (Prescription only)[1]
  • EU: Rx-only
  • In general: ℞ (Prescription only)
Identifiers
  • all-P-ambo-2'-O-(2-Methoxyethyl)-P-thioadenylyl-(3'→5')-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'→5')-2'-O-(2- methoxyethyl)-5-methyl-P-thiouridylyl-(3'→5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'→5')-P-thiothymidylyl-(3'→5')-P- thiothymidylyl-(3'→5')-2'-deoxy-P-thioguanylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'→5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-deoxy-P-thioguanylyl-(3'→5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'→5')-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyluridine[2]
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
Chemical and physical data
FormulaC230H320N63O125P19S19
Molar mass7165.05 g·mol−1
  • Cc1cn(c(=O)[nH]c1=O)[C@H]2C[C@@H]([C@H](O2)COP(=O)(O[C@H]3C[C@@H](O[C@@H]3COP(=O)(O[C@H]4C[C@@H](O[C@@H]4COP(=O)(O[C@@H]5[C@H](O[C@H]([C@@H]5OCCOC)n6cc(c(=O)[nH]c6=O)C)COP(=O)(O[C@@H]7[C@H](O[C@H]([C@@H]7OCCOC)n8cc(c(=O)[nH]c8=O)C)COP(=O)(O[C@@H]9[C@H](O[C@H]([C@@H]9OCCOC)n1cc(c(nc1=O)N)C)COP(=O)(O[C@@H]1[C@H](O[C@H]([C@@H]1OCCOC)n1cnc2c1nc([nH]c2=O)N)COP(=O)(O[C@@H]1[C@H](O[C@H]([C@@H]1OCCOC)n1cnc2c1ncnc2N)CO)S)S)S)S)S)n1cc(c(nc1=O)N)C)S)n1cc(c(=O)[nH]c1=O)C)S)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cnc2c1nc([nH]c2=O)N)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cc(c(=O)[nH]c1=O)C)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cc(c(nc1=O)N)C)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cc(c(nc1=O)N)C)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cnc2c1ncnc2N)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cnc2c1nc([nH]c2=O)N)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cc(c(nc1=O)N)C)OP(=O)(OC[C@@H]1[C@H]([C@H]([C@@H](O1)n1cc(c(=O)[nH]c1=O)C)OCCOC)OP(=O)(OC[C@@H]1[C@H]([C@H]([C@@H](O1)n1cc(c(=O)[nH]c1=O)C)OCCOC)OP(=O)(OC[C@@H]1[C@H]([C@H]([C@@H](O1)n1cc(c(=O)[nH]c1=O)C)OCCOC)OP(=O)(OC[C@@H]1[C@H]([C@H]([C@@H](O1)n1cnc2c1ncnc2N)OCCOC)OP(=O)(OC[C@@H]1[C@H]([C@H]([C@@H](O1)n1cc(c(=O)[nH]c1=O)C)OCCOC)O)S)S)S)S)S)S)S)S)S)S)S)S
  • InChI=1S/C230H320N63O125P19S19/c1-98-55-274(217(308)254-178(98)231)142-45-112(400-419(322,438)361-70-124-113(46-143(381-124)275-56-99(2)179(232)255-218(275)309)401-423(326,442)367-76-130-119(52-149(387-130)288-92-248-152-183(236)242-89-245-186(152)288)407-427(330,446)369-78-132-121(54-151(389-132)290-94-252-156-190(290)260-215(240)263-202(156)306)408-425(328,444)365-72-125-114(47-144(382-125)276-57-100(3)180(233)256-219(276)310)406-428(331,447)372-81-134-160(171(354-38-28-344-18)206(393-134)283-64-107(10)196(300)269-226(283)317)412-432(335,451)375-84-137-163(173(356-40-30-346-20)208(395-137)285-66-109(12)198(302)271-228(285)319)415-435(338,454)377-86-139-165(175(358-42-32-348-22)210(397-139)287-68-111(14)200(304)273-230(287)321)416-436(339,455)379-87-140-166(176(359-43-33-349-23)212(398-140)292-96-250-154-185(238)244-91-247-188(154)292)417-430(333,449)371-80-133-158(295)168(351-35-25-341-15)204(391-133)282-63-106(9)195(299)268-225(282)316)123(380-142)71-362-421(324,440)403-117-50-147(279-61-104(7)193(297)266-223(279)314)386-128(117)75-366-426(329,445)409-120-53-150(289-93-251-155-189(289)259-214(239)262-201(155)305)388-131(120)77-368-424(327,443)405-118-51-148(280-62-105(8)194(298)267-224(280)315)385-127(118)74-364-422(325,441)404-116-49-146(278-60-103(6)192(296)265-222(278)313)384-126(116)73-363-420(323,439)402-115-48-145(277-58-101(4)181(234)257-220(277)311)383-129(115)79-370-429(332,448)411-162-136(394-207(172(162)355-39-29-345-19)284-65-108(11)197(301)270-227(284)318)83-374-434(337,453)414-164-138(396-209(174(164)357-41-31-347-21)286-67-110(13)199(303)272-229(286)320)85-376-433(336,452)413-161-135(392-205(170(161)353-37-27-343-17)281-59-102(5)182(235)258-221(281)312)82-378-437(340,456)418-167-141(399-213(177(167)360-44-34-350-24)293-97-253-157-191(293)261-216(241)264-203(157)307)88-373-431(334,450)410-159-122(69-294)390-211(169(159)352-36-26-342-16)291-95-249-153-184(237)243-90-246-187(153)291/h55-68,89-97,112-151,158-177,204-213,294-295H,25-54,69-88H2,1-24H3,(H,322,438)(H,323,439)(H,324,440)(H,325,441)(H,326,442)(H,327,443)(H,328,444)(H,329,445)(H,330,446)(H,331,447)(H,332,448)(H,333,449)(H,334,450)(H,335,451)(H,336,452)(H,337,453)(H,338,454)(H,339,455)(H,340,456)(H2,231,254,308)(H2,232,255,309)(H2,233,256,310)(H2,234,257,311)(H2,235,258,312)(H2,236,242,245)(H2,237,243,246)(H2,238,244,247)(H,265,296,313)(H,266,297,314)(H,267,298,315)(H,268,299,316)(H,269,300,317)(H,270,301,318)(H,271,302,319)(H,272,303,320)(H,273,304,321)(H3,239,259,262,305)(H3,240,260,263,306)(H3,241,261,264,307)/t112-,113-,114-,115-,116-,117-,118-,119-,120-,121-,122+,123+,124+,125+,126+,127+,128+,129+,130+,131+,132+,133+,134+,135+,136+,137+,138+,139+,140+,141+,142+,143+,144+,145+,146+,147+,148+,149+,150+,151+,158+,159+,160+,161+,162+,163+,164+,165+,166+,167+,168+,169+,170+,171+,172+,173+,174+,175+,176+,177+,204+,205+,206+,207+,208+,209+,210+,211+,212+,213+,419?,420?,421?,422?,423?,424?,425?,426?,427?,428?,429?,430?,431?,432?,433?,434?,435?,436?,437?/m0/s1
  • Key:IJUQCWMZCMFFJP-GQSLRNSLSA-N

Volanesorsen, sold under the brand name Waylivra, is a triglyceride-reducing drug. It is a second-generation[3] 2'-O-methoxyethyl (2'-MOE) chimeric antisense therapeutic oligonucleotide (ASO)[4] that targets the messenger RNA for apolipoprotein C3 (apo-CIII).

The most common side effects include reduced platelet levels and reactions at the site of the injection such as pain, swelling, itching, or bruising.[5]

Volanesorsen, is an 'antisense oligonucleotide,' a very short piece of synthetic RNA (a type of genetic material).[5] It has been designed to block the production of a protein that slows down the breakdown of fats called apolipoprotein C-III.[5] By blocking the production of this protein, the medicine reduces the level of triglycerides in the blood and, as a result, fat accumulation in the body, which is expected to reduce the risk of pancreatitis.[5]

  1. ^ "Waylivra (volanesorsen) 285 mg solution for injection in pre-filled syringe - Summary of Product Characteristics (SmPC)". (emc). 28 August 2019. Retrieved 19 May 2020.
  2. ^ Cite error: The named reference INN was invoked but never defined (see the help page).
  3. ^ Pechlaner R, Tsimikas S, Yin X, Willeit P, Baig F, Santer P, et al. (February 2017). "Very-Low-Density Lipoprotein-Associated Apolipoproteins Predict Cardiovascular Events and Are Lowered by Inhibition of APOC-III". Journal of the American College of Cardiology. 69 (7): 789–800. doi:10.1016/j.jacc.2016.11.065. PMC 5314136. PMID 28209220.
  4. ^ Graham MJ, Lee RG, Bell TA, Fu W, Mullick AE, Alexander VJ, et al. (May 2013). "Antisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, nonhuman primates, and humans". Circulation Research. 112 (11): 1479–90. doi:10.1161/CIRCRESAHA.111.300367. PMID 23542898.
  5. ^ a b c d "Waylivra EPAR". European Medicines Agency (EMA). 24 September 2018. Retrieved 19 May 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.