Homozygous Familial Hypercholesterolemia

Also known as: Homozygous familial hypercholesterolemia / Familial homozygous hypercholesterolemia / Homozygous familial hypercholesterolaemia / Type IIa hyperlipoproteinemia / Hyperlipoproteinemia Type IIa / Fredrickson Type IIa hyperlipoproteinemia / Type IIa hyperlipoproteinaemia / Familial hypercholesterolemia / Fredrickson Type IIa hyperlipoproteinaemia / Fredrickson Type IIa hyperlipidemia / Fredrickson Type IIa hyperlipidaemia / Fredrickson Type IIa lipidemia / Fredrickson Type IIa lipidaemia / Type IIa hyperlipidaemia / Type IIa hyperlipidemia

DrugDrug NameDrug Description
DB01076AtorvastatinAn HMG-CoA reductase inhibitor used to lower lipid levels and reduce the risk of cardiovascular disease including myocardial infarction and stroke.
DB09303EvolocumabA PCSK9 (proprotein convertase subtilisin kexin type 9) inhibitor antibody used as an adjunct to LDL cholesterol reducing therapies, aiding in the prevention of cardiovascular events and cardiovascular revascularization procedures.
DB00973EzetimibeA cholesterol absorption inhibitor used to lower total cholesterol, LDL-C, Apo B, and non-HDL-C in primary hyperlipidemia and familial cholesterolemia.
DB08827LomitapideA microsomal triglyceride transfer protein inhibitor used to lower cholesterol associated with homozygous familial hypercholesterolemia (HoFH), reducing risk of cardiovascular events such as myocardial infarction and stroke.
DB05528MipomersenAn oligonucleotide drug used for the treatment of homozygous familial hypercholesterolemia.
DB01098RosuvastatinAn HMG-CoA reductase inhibitor used to lower lipid levels and reduce the risk of cardiovascular disease including myocardial infarction and stroke.
DB00641SimvastatinAn HMG-CoA reductase inhibitor used to lower lipid levels and reduce the risk of cardiovascular events including myocardial infarction and stroke.
DrugDrug NameTargetType
DB01076Atorvastatin3-hydroxy-3-methylglutaryl-coenzyme A reductasetarget
DB01076AtorvastatinCytochrome P450 3A4enzyme
DB01076AtorvastatinDipeptidyl peptidase 4target
DB01076AtorvastatinCytochrome P450 2D6enzyme
DB01076AtorvastatinCytochrome P450 2C9enzyme
DB01076AtorvastatinCytochrome P450 2C19enzyme
DB01076AtorvastatinCytochrome P450 3A5enzyme
DB01076AtorvastatinCytochrome P450 3A7enzyme
DB01076AtorvastatinP-glycoprotein 1transporter
DB01076AtorvastatinSolute carrier organic anion transporter family member 1A2transporter
DB01076AtorvastatinSolute carrier organic anion transporter family member 1B1transporter
DB01076AtorvastatinMultidrug resistance-associated protein 4transporter
DB01076AtorvastatinMultidrug resistance-associated protein 5transporter
DB01076AtorvastatinMultidrug resistance-associated protein 1transporter
DB01076AtorvastatinCytochrome P450 2B6enzyme
DB01076AtorvastatinCytochrome P450 2C8enzyme
DB01076AtorvastatinAryl hydrocarbon receptortarget
DB01076AtorvastatinSolute carrier organic anion transporter family member 2B1transporter
DB01076AtorvastatinSolute carrier organic anion transporter family member 1B3transporter
DB01076AtorvastatinUDP-glucuronosyltransferase 1-1enzyme
DB01076AtorvastatinUDP-glucuronosyltransferase 1-3enzyme
DB01076AtorvastatinCanalicular multispecific organic anion transporter 1transporter
DB01076AtorvastatinBile salt export pumptransporter
DB01076AtorvastatinSerum albumincarrier
DB01076AtorvastatinHistone deacetylase 2target
DB01076AtorvastatinNuclear receptor subfamily 1 group I member 3target
DB09303EvolocumabProprotein convertase subtilisin/kexin type 9enzyme
DB00973EzetimibeNiemann-Pick C1-like protein 1target
DB00973EzetimibeAminopeptidase Ntarget
DB00973EzetimibeSterol O-acyltransferase 1target
DB00973EzetimibeUDP-glucuronosyltransferase 1-1enzyme
DB00973EzetimibeUDP-glucuronosyltransferase 1-3enzyme
DB00973EzetimibeP-glycoprotein 1transporter
DB00973EzetimibeCanalicular multispecific organic anion transporter 1transporter
DB00973EzetimibeSolute carrier organic anion transporter family member 1B1transporter
DB00973EzetimibeUDP-glucuronosyltransferase 2B15enzyme
DB00973EzetimibeUDP-glucuronosyltransferase 2B7enzyme
DB00973EzetimibeCytochrome P450 3A4enzyme
DB00973EzetimibeATP-binding cassette sub-family G member 5transporter
DB00973EzetimibeCanalicular multispecific organic anion transporter 2transporter
DB00973EzetimibeATP-binding cassette sub-family G member 2transporter
DB00973EzetimibeATP-binding cassette sub-family G member 8transporter
DB00973EzetimibeCytochrome P450 2C8enzyme
DB00973EzetimibeBile salt export pumptransporter
DB08827LomitapideMicrosomal triglyceride transfer protein large subunittarget
DB08827LomitapideCytochrome P450 3A4enzyme
DB08827LomitapideP-glycoprotein 1transporter
DB05528MipomersenmRNA of ApoB-100target
DB01098Rosuvastatin3-hydroxy-3-methylglutaryl-coenzyme A reductasetarget
DB01098RosuvastatinCytochrome P450 2C9enzyme
DB01098RosuvastatinMultidrug resistance-associated protein 4transporter
DB01098RosuvastatinSolute carrier organic anion transporter family member 1A2transporter
DB01098RosuvastatinSolute carrier organic anion transporter family member 1B3transporter
DB01098RosuvastatinSolute carrier organic anion transporter family member 2B1transporter
DB01098RosuvastatinCystine/glutamate transportertransporter
DB01098RosuvastatinSolute carrier organic anion transporter family member 1B1transporter
DB01098RosuvastatinBile salt export pumptransporter
DB01098RosuvastatinATP-binding cassette sub-family G member 2transporter
DB01098RosuvastatinSerum albumincarrier
DB01098RosuvastatinCanalicular multispecific organic anion transporter 1transporter
DB01098RosuvastatinSolute carrier family 22 member 8transporter
DB01098RosuvastatinSodium/bile acid cotransportertransporter
DB01098RosuvastatinIntegrin alpha-Ltarget
DB00641Simvastatin3-hydroxy-3-methylglutaryl-coenzyme A reductasetarget
DB00641SimvastatinCytochrome P450 3A4enzyme
DB00641SimvastatinCytochrome P450 2C8enzyme
DB00641SimvastatinCytochrome P450 2C9enzyme
DB00641SimvastatinCytochrome P450 3A5enzyme
DB00641SimvastatinP-glycoprotein 1transporter
DB00641SimvastatinSolute carrier organic anion transporter family member 1A2transporter
DB00641SimvastatinSolute carrier organic anion transporter family member 1B1transporter
DB00641SimvastatinCytochrome P450 2B6enzyme
DB00641SimvastatinCytochrome P450 2D6enzyme
DB00641SimvastatinUDP-glucuronosyltransferase 1-1enzyme
DB00641SimvastatinUDP-glucuronosyltransferase 1-3enzyme
DB00641SimvastatinUDP-glucuronosyltransferase 2B7enzyme
DB00641SimvastatinCanalicular multispecific organic anion transporter 1transporter
DB00641SimvastatinIntegrin alpha-Ltarget
DB00641SimvastatinBile salt export pumptransporter
DB00641SimvastatinCytochrome P450 2C19enzyme
DB00641SimvastatinSolute carrier organic anion transporter family member 2B1transporter
DB00641SimvastatinSolute carrier organic anion transporter family member 1B3transporter
DB00641SimvastatinHistone deacetylase 2target
DrugDrug NamePhaseStatusCount
DB15354Evinacumab2Completed1
DB00973Ezetimibe2Recruiting1
DB08827Lomitapide2Completed1
DB12390MBX-80252Completed1
DB09303Evolocumab2 / 3Completed1
DB09302Alirocumab3Recruiting1
DB06630Anacetrapib3Terminated1
DB15354Evinacumab3Active Not Recruiting1
DB15354Evinacumab3Enrolling by Invitation1
DB09303Evolocumab3Enrolling by Invitation1
DB14901Inclisiran3Recruiting1
DB08827Lomitapide3Completed1
DB08827Lomitapide3Withdrawn1
DB00973EzetimibeNot AvailableCompleted1
DB08827LomitapideNot AvailableEnrolling by Invitation1
DB08827LomitapideNot AvailableWithdrawn1