Heterozygous Familial Hypercholesterolemia (HeFH)

Also known as: Familial Hypercholesterolemia - Heterozygous / Heterozygous Familial Hypercholesterolaemia / Heterozygous Familial Hypercholesterolemia / Familial hypercholesterolemia - heterozygous (disorder) / Pure hypercholesterolemia, unspecified / Type IIa hyperlipoproteinemia / Hyperlipoproteinemia Type IIa / Fredrickson Type IIa hyperlipoproteinemia / Fredrickson type IIa hyperlipoproteinemia (disorder) / Type IIa hyperlipoproteinaemia / 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
DB00945Acetylsalicylic acidA salicylate used to treat pain, fever, inflammation, migraines, and reducing the risk of major adverse cardiovascular events.
DB09302AlirocumabA PCSK9 inhibitor used as an adjunct to manage heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease in patients who require additional lowering of LDL-cholesterol (LDL-C).
DB01076AtorvastatinAn HMG-CoA reductase inhibitor used to lower lipid levels and reduce the risk of cardiovascular disease including myocardial infarction and stroke.
DB11936Bempedoic acidA drug used in conjunction with lifestyle modification and/or other agents for the treatment of refractory hypercholesterolemia.
DB00930ColesevelamA bile acid sequestrant used to lower LDL-C in adults with hyperlipidemia and pediatric patients with heterozygous familial hypercholesterolemia, and to improve glycemic control in type 2 diabetes.
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.
DB01095FluvastatinAn HMG-CoA reductase inhibitor used to lower lipid levels and reduce the risk of cardiovascular disease including myocardial infarction and stroke.
DB14901InclisiranA PCSK9-targeting short interfering RNA (siRNA) that lowers plasma LDL-cholesterol levels.
DB00227LovastatinAn HMG-CoA reductase inhibitor used to lower LDL cholesterol and reduce the risk of cardiovascular disease and associated conditions, including myocardial infarction and stroke.
DB08860PitavastatinAn HMG-CoA reductase inhibitor used to lower lipid levels and reduce the risk of cardiovascular disease including myocardial infarction and stroke.
DB00175PravastatinAn HMG-CoA reductase inhibitor used to lower lipid levels and to reduce the risk of cardiovascular events, including myocardial infarction and stroke.
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
DB00945Acetylsalicylic acidProstaglandin G/H synthase 1target
DB00945Acetylsalicylic acidProstaglandin G/H synthase 2target
DB00945Acetylsalicylic acidAldo-keto reductase family 1 member C1target
DB00945Acetylsalicylic acidSolute carrier family 22 member 6transporter
DB00945Acetylsalicylic acidP-glycoprotein 1transporter
DB00945Acetylsalicylic acidCytochrome P450 2C19enzyme
DB00945Acetylsalicylic acidCytochrome P450 2C9enzyme
DB00945Acetylsalicylic acid5'-AMP-activated protein kinasetarget
DB00945Acetylsalicylic acidEndothelin-1 receptortarget
DB00945Acetylsalicylic acidCellular tumor antigen p53target
DB00945Acetylsalicylic acid78 kDa glucose-regulated proteintarget
DB00945Acetylsalicylic acidRibosomal protein S6 kinase alpha-3target
DB00945Acetylsalicylic acidNF-kappa-B inhibitor alphatarget
DB00945Acetylsalicylic acidTumor necrosis factor-inducible gene 6 proteintarget
DB00945Acetylsalicylic acidCaspase-1target
DB00945Acetylsalicylic acidCaspase-3target
DB00945Acetylsalicylic acidInhibitor of nuclear factor kappa-B kinase subunit betatarget
DB00945Acetylsalicylic acidExtracellular signal-regulated kinase (ERK)target
DB00945Acetylsalicylic acidG1/S-specific cyclin-D1target
DB00945Acetylsalicylic acidMyc proto-oncogene proteintarget
DB00945Acetylsalicylic acidProliferating cell nuclear antigentarget
DB00945Acetylsalicylic acidCyclin Atarget
DB00945Acetylsalicylic acidUDP-glucuronosyltransferase 1-6enzyme
DB00945Acetylsalicylic acidArylamine N-acetyltransferase 2enzyme
DB00945Acetylsalicylic acidSolute carrier family 22 member 8transporter
DB00945Acetylsalicylic acidSialidase-1target
DB09302AlirocumabProprotein convertase subtilisin/kexin type 9target
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
DB11936Bempedoic acidATP-citrate synthasetarget
DB11936Bempedoic acidVery long-chain acyl-CoA synthetaseenzyme
DB11936Bempedoic acidSolute carrier organic anion transporter family member 1B1transporter
DB11936Bempedoic acidCanalicular multispecific organic anion transporter 2transporter
DB11936Bempedoic acidSolute carrier organic anion transporter family member 1B3transporter
DB11936Bempedoic acidUDP-glucuronosyltransferase 2B7enzyme
DB11936Bempedoic acidNUAK family SNF1-like kinase 2enzyme
DB00930ColesevelamBile acidstarget
DB09303EvolocumabProprotein convertase subtilisin/kexin type 9target
DB01095Fluvastatin3-hydroxy-3-methylglutaryl-coenzyme A reductasetarget
DB01095FluvastatinCytochrome P450 2C9enzyme
DB01095FluvastatinCytochrome P450 3A4enzyme
DB01095FluvastatinCytochrome P450 2C8enzyme
DB01095FluvastatinCytochrome P450 2D6enzyme
DB01095FluvastatinCytochrome P450 2C19enzyme
DB01095FluvastatinCytochrome P450 1A1enzyme
DB01095FluvastatinSolute carrier organic anion transporter family member 1B1transporter
DB01095FluvastatinSolute carrier organic anion transporter family member 1B3transporter
DB01095FluvastatinSolute carrier organic anion transporter family member 2B1transporter
DB01095FluvastatinSolute carrier family 15 member 1transporter
DB01095FluvastatinCytochrome P450 3A5enzyme
DB01095FluvastatinUDP-glucuronosyltransferase 1-1enzyme
DB01095FluvastatinUDP-glucuronosyltransferase 1-3enzyme
DB01095FluvastatinUDP-glucuronosyltransferase 2B7enzyme
DB01095FluvastatinCanalicular multispecific organic anion transporter 1transporter
DB01095FluvastatinCytochrome P450 2B6enzyme
DB01095FluvastatinHistone deacetylase 2target
DB14901InclisiranProprotein convertase subtilisin/kexin type 9target
DB00227Lovastatin3-hydroxy-3-methylglutaryl-coenzyme A reductasetarget
DB00227LovastatinCytochrome P450 3A4enzyme
DB00227LovastatinCytochrome P450 2C8enzyme
DB00227LovastatinIntegrin alpha-Ltarget
DB00227LovastatinHistone deacetylase 2target
DB00227LovastatinSerum paraoxonase/lactonase 3enzyme
DB00227LovastatinP-glycoprotein 1transporter
DB00227LovastatinSolute carrier organic anion transporter family member 1A2transporter
DB00227LovastatinSolute carrier organic anion transporter family member 1B1transporter
DB00227LovastatinUDP-glucuronosyltransferase 1-1enzyme
DB00227LovastatinUDP-glucuronosyltransferase 1-3enzyme
DB00227LovastatinUDP-glucuronosyltransferase 2B7enzyme
DB00227LovastatinCanalicular multispecific organic anion transporter 1transporter
DB00227LovastatinBile salt export pumptransporter
DB00227LovastatinSerum albumincarrier
DB00227LovastatinCytochrome P450 2C19enzyme
DB00227LovastatinSolute carrier organic anion transporter family member 2B1transporter
DB00227LovastatinSolute carrier organic anion transporter family member 1B3transporter
DB08860PitavastatinSolute carrier organic anion transporter family member 1B1transporter
DB08860PitavastatinSolute carrier organic anion transporter family member 1B3transporter
DB08860PitavastatinUDP-glucuronosyltransferase 2B7enzyme
DB08860PitavastatinUDP-glucuronosyltransferase 1-3enzyme
DB08860PitavastatinCytochrome P450 2C9enzyme
DB08860PitavastatinCytochrome P450 2C8enzyme
DB08860Pitavastatin3-hydroxy-3-methylglutaryl-coenzyme A reductasetarget
DB08860PitavastatinSolute carrier organic anion transporter family member 2B1transporter
DB08860PitavastatinSodium/bile acid cotransportertransporter
DB08860PitavastatinATP-binding cassette sub-family G member 2transporter
DB08860PitavastatinP-glycoprotein 1transporter
DB08860PitavastatinCanalicular multispecific organic anion transporter 1transporter
DB08860PitavastatinSerum albumincarrier
DB08860PitavastatinAlpha-1-acid glycoprotein 1carrier
DB08860PitavastatinBile salt export pumptransporter
DB08860PitavastatinIntegrin alpha-Ltarget
DB00175Pravastatin3-hydroxy-3-methylglutaryl-coenzyme A reductasetarget
DB00175PravastatinSolute carrier organic anion transporter family member 2B1transporter
DB00175PravastatinP-glycoprotein 1transporter
DB00175PravastatinSolute carrier organic anion transporter family member 1A2transporter
DB00175PravastatinSolute carrier family 22 member 6transporter
DB00175PravastatinSolute carrier family 22 member 8transporter
DB00175PravastatinCanalicular multispecific organic anion transporter 1transporter
DB00175PravastatinSolute carrier family 22 member 11transporter
DB00175PravastatinATP-binding cassette sub-family G member 2transporter
DB00175PravastatinSolute carrier family 22 member 7transporter
DB00175PravastatinSolute carrier organic anion transporter family member 1B1transporter
DB00175PravastatinMonocarboxylate transporter 1transporter
DB00175PravastatinBile salt export pumptransporter
DB00175PravastatinSolute carrier organic anion transporter family member 1B3transporter
DB00175PravastatinHistone deacetylase 2target
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
DB00641SimvastatinCocaine esteraseenzyme
DB00641SimvastatinLiver carboxylesterase 1enzyme
DrugDrug NamePhaseStatusCount
DB11629Laropiprant1Terminated1
DB00627Niacin1Terminated1
DB09302Alirocumab2Completed1
DB18538CER-0012Completed1
DB00973Ezetimibe2Completed1
DB14901Inclisiran2Completed1
DB12914Resmetirom2Completed1
DB09302Alirocumab3Completed2
DB06630Anacetrapib3Completed1
DB01076Atorvastatin3Completed1
DB12456Bococizumab3Completed1
DB05035Eprotirome3Terminated1
DB09303Evolocumab3Completed1
DB01095Fluvastatin3Completed1
DB14901Inclisiran3Active Not Recruiting1
DB14901Inclisiran3Completed2
DB05528Mipomersen3Completed1
DB01098Rosuvastatin3Completed1
DB00641Simvastatin3Completed1
DB16336Tafolecimab3Completed1
DB09302Alirocumab4Terminated1