Table 1. Novel pharmacological interventions for LDL-lowering.
| Target | Deficiency or carriers of PTV | Compounds | Randomized controlled trials | Mendelian randomization |
|---|---|---|---|---|
| NPCL1 | Heterozygous carriers (1 in 650 individuals) | Ezetimibe | IMPROVE-IT | Ref 20 |
| PCSK9 | Familial hypobetalipoproteinemia | Evolocumab Alirocumab |
FOURIER ODYSSEY OUTCOMES |
Ref 23 |
| MTTP | Abetalipoproteinemia | Lomitapide | NA | NA |
| APOB | Familial hypobetalipoproteinemia | Mipomersen | NA | Ref 5 |
| ANGPTL3 | Familial combined hypolipoproteinemia | Evinacumab | NA | Ref 22 |
| ACLY | NA | Bempedoic Acid | NA | Ref 21 |
NPC1L1, Niemann-Pick C1-Like 1; PCSK9, proprotein convertase subtilisin-kexin type 9; MTTP, microsomal triglyceride transfer protein; APOB, apolipoprotein B; ANGPTL3, Angiopoietin-like 3; Ref, reference, NA, not available; ACLY, ATP citrate lyase.