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. 2021 Oct 22;43(4):611–653. doi: 10.1210/endrev/bnab037

Table 10.

Emerging treatments

Class Agent Dose Mechanism of action Main indication Comments
ANGPTL3 inhibitors Evinacumab 15 mg/kg IV every 4 weeks Global lipid reduction LDL-C reduction in HoFH; TG reduction ↓ LDL-C by 30%-50% and ↓ TG by 25%-45%
Vupanorsen 40-80 mg SC every 4 weeks Global lipid reduction LDL-C reduction in HoFH; TG reduction ↓ LDL-C by 30%-50% and ↓ TG by 25%-45%
AROANG3 50-200 mg SC every 2-4 weeks Global lipid reduction LDL-C reduction in HoFH; TG reduction ↓ LDL-C by 30%-50% and ↓ TG by 25%-45%
Apo C-III inhibitors Volanesorsen 300 mg SC every 1 week ASO knocks down apo C-III TG reduction ↓ TG by 50%-70%
AKCEA-apoC3-LRX Undetermined ASO knocks down apo C-III TG reduction ↓ TG by 50%-70%; GalNAc linked
AROAPOC3 50 mg SC every 12 weeks siRNA knocks down apo C-III TG reduction ↓ TG by 50%-70%
anti-Lp(a) Olpasiran Undetermined siRNA knocks down Lp(a) Lp(a) reduction ↓ Lp(a) by 50%-70%
Pelacarsen 80 mg SC every 4 weeks ASO knocks down Lp(a) Lp(a) reduction ↓ Lp(a) by 50%-70%
Other Gemcabene 300-900 mg/d Unclear LDL-C reduction ↓ LDL-C by 15%-25% as monotherapy or added to statin

Abbreviations: ANGPTL3, angiopoietin-like protein 3; apo, apolipoprotein; ASO, antisense oligonucleotide; GalNAc, conjugated with N-acetylgalactosamine; HoFH, homozygous familial hypercholesteremia; LDL-C, low-density lipoprotein cholesterol; Lp(a), lipoprotein(a); SC, subcutaneous; siRNA, small interfering RNA; TG, triglyceride.