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Mipomersen

CHEMBL2219536 Phase 4 Approuvé Oligonucleotide
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

This antisense oligonucleotide therapy targets the mRNA for apolipoprotein B-100 in liver cells, dramatically reducing production of LDL (bad) cholesterol and other harmful lipoproteins in patients with a severe inherited cholesterol disorder called familial hypercholesterolemia. It is injected subcutaneously once weekly for patients who cannot achieve adequate cholesterol control with standard therapies.

Aires thérapeutiques

Hyperlipidemia

Mécanisme d'action

Binds to complementary mRNA sequences through Watson-Crick base pairing, modulating gene expression by promoting mRNA degradation via RNase H or altering pre-mRNA splicing.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Mécanisme

Binds to complementary mRNA sequences through Watson-Crick base pairing, modulating gene expression by promoting mRNA degradation via RNase H or altering pre-mRNA splicing.

Structure 2D

SVG PNG

Cite this structure


                        

                            
                            
                        

                    

                


                
                

                    

                    

                        
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SMILES

                    
COCCO[C@@H]1[C@H](SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(=O)[nH]c3=O)[C@H](OCCOC)[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(N)nc3=O)[C@H](OCCOC)[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cnc4c(N)ncnc43)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cnc4c(=O)[nH]c(N)nc43)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(=O)[nH]c3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(N)nc3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(=O)[nH]c3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cnc4c(=O)[nH]c(N)nc43)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(N)nc3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(=O)[nH]c3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(=O)[nH]c3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cc(C)c(N)nc3=O)C[C@@H]2SP(=O)(O)OC[C@H]2O[C@@H](n3cnc4c(=O)[nH]c(N)nc43)[C@H](OCCOC)[C@@H]2SP(=O)(O)OC[C@@H]2O[C@H](n3cc(C)c(N)nc3=O)[C@@H](OCCOC)[C@H]2SP(=O)(O)OC[C@@H]2O[C@H](n3cnc4c(N)ncnc43)[C@@H](OCCOC)[C@H]2SP(=O)(O)OC[C@@H]2O[C@H](n3cc(C)c(N)nc3=O)[C@@H](OCCOC)[C@H]2SP(=O)(O)OC[C@@H]2O[C@H](n3cc(C)c(N)nc3=O)[C@@H](OCCOC)[C@H]2O)[C@@H](COP(=O)(O)S[C@H]2[C@@H](OCCOC)[C@H](n3cc(C)c(N)nc3=O)O[C@@H]2COP(=O)(O)S[C@H]2[C@@H](OCCOC)[C@H](n3cnc4c(=O)[nH]c(N)nc43)O[C@@H]2CO)O[C@H]1n1cc(C)c(N)nc1=O

                

                

                
                

                    
InChI

                    
InChI=1S/C230H324N67O122P19S19/c1-97-55-278(217(309)256-177(97)231)141-45-131(112(400-141)70-381-422(327,328)442-136-50-146(283-66-108(12)196(302)275-228(283)320)406-118(136)76-387-427(337,338)448-140-54-150(294-93-253-154-191(294)266-214(243)270-200(154)306)409-120(140)78-389-428(339,340)446-138-52-148(292-91-250-151-186(240)246-89-248-188(151)292)407-121(138)79-390-430(343,344)450-169-124(413-204(160(169)373-37-27-363-17)286-59-101(5)181(235)260-221(286)313)83-394-435(353,354)455-174-128(417-209(165(174)378-42-32-368-22)291-68-110(14)198(304)277-230(291)322)87-396-434(351,352)453-172-126(415-207(163(172)376-40-30-366-20)289-62-104(8)184(238)263-224(289)316)85-395-433(349,350)452-171-123(412-206(162(171)375-39-29-365-19)288-61-103(7)183(237)262-223(288)315)82-393-432(347,348)449-168-111(69-298)410-211(159(168)372-36-26-362-16)296-95-254-155-192(296)267-215(244)271-201(155)307)439-420(323,324)384-74-117-137(51-147(405-117)284-67-109(13)197(303)276-229(284)321)444-425(333,334)388-77-119-139(53-149(408-119)293-92-252-153-190(293)265-213(242)269-199(153)305)447-426(335,336)386-71-113-132(46-142(401-113)279-56-98(2)178(232)257-218(279)310)440-421(325,326)383-73-115-135(49-145(403-115)282-65-107(11)195(301)274-227(282)319)443-424(331,332)385-75-116-134(48-144(404-116)281-64-106(10)194(300)273-226(281)318)441-423(329,330)382-72-114-133(47-143(402-114)280-57-99(3)179(233)258-219(280)311)445-429(341,342)392-81-129-176(167(380-44-34-370-24)212(419-129)297-96-255-156-193(297)268-216(245)272-202(156)308)457-438(359,360)398-86-127-173(164(377-41-31-367-21)208(416-127)290-63-105(9)185(239)264-225(290)317)454-436(355,356)399-88-130-175(166(379-43-33-369-23)210(418-130)295-94-251-152-187(241)247-90-249-189(152)295)456-437(357,358)397-84-125-170(161(374-38-28-364-18)205(414-125)287-60-102(6)182(236)261-222(287)314)451-431(345,346)391-80-122-157(299)158(371-35-25-361-15)203(411-122)285-58-100(4)180(234)259-220(285)312/h55-68,89-96,111-150,157-176,203-212,298-299H,25-54,69-88H2,1-24H3,(H,323,324)(H,325,326)(H,327,328)(H,329,330)(H,331,332)(H,333,334)(H,335,336)(H,337,338)(H,339,340)(H,341,342)(H,343,344)(H,345,346)(H,347,348)(H,349,350)(H,351,352)(H,353,354)(H,355,356)(H,357,358)(H,359,360)(H2,231,256,309)(H2,232,257,310)(H2,233,258,311)(H2,234,259,312)(H2,235,260,313)(H2,236,261,314)(H2,237,262,315)(H2,238,263,316)(H2,239,264,317)(H2,240,246,248)(H2,241,247,249)(H,273,300,318)(H,274,301,319)(H,275,302,320)(H,276,303,321)(H,277,304,322)(H3,242,265,269,305)(H3,243,266,270,306)(H3,244,267,271,307)(H3,245,268,272,308)/t111-,112-,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-,157+,158+,159-,160-,161+,162-,163-,164+,165-,166+,167-,168-,169-,170+,171-,172-,173+,174-,175+,176-,203+,204-,205+,206-,207-,208+,209-,210+,211-,212-/m1/s1

                

                

                

                    
                    

                        
HBD / HBA

                        
- / -

                    

                    
                    
                

            


            
            

                
                


    
    
        
    
    
    
No targets recorded

    
    
Target interaction data is not yet available for this drug.

    



                
            


            
            

                
                


    
    
        
    
    
    
No interactions recorded

    
    
Drug interaction data is not yet available for this compound.

    



                
            


            
            

                
                


    
    
        
    
    
    
No side effects recorded

    
    
Side effect data is not yet available for this drug.

    



                
            


            
            
            

                
Foire aux questions

                

                    
                    

                        
                        

                            
This antisense oligonucleotide therapy targets the mRNA for apolipoprotein B-100 in liver cells, dramatically reducing production of LDL (bad) cholesterol and other harmful lipoproteins in patients with a severe inherited cholesterol disorder called familial hypercholesterolemia. It is injected subcutaneously once weekly for patients who cannot achieve adequate cholesterol control with standard therapies.

                        

                    

                    

                    

                    
                    

                        
                        

                            
Binds to complementary mRNA sequences through Watson-Crick base pairing, modulating gene expression by promoting mRNA degradation via RNase H or altering pre-mRNA splicing.

                        

                    

                    

                    

                    

                        
                        

                            
Yes, Mipomersen is an approved drug. It has reached clinical phase 4. It is classified as a Oligonucleotide.

                        

                    

                

            

            

            
            

            
            

            
            
{# References & Data Sources section for drug detail pages.
   Renders standard pharmacological database links plus the drug's data_sources field. #}


    
References & Data Sources

    
    
        
        
  • 
            
            
                ChEMBL — European Bioinformatics Institute (EBI).
                CHEMBL2219536.
                Open-access bioactivity database.
            
        
    • 
        
        
        
        
        
    

    
Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.




        


        
        
    


    



    
Avertissement médical

    

        This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.
    

    

        Data sources:
        ChEMBL,
        PubChem,
        DailyMed.