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.