Inclisiran, a small interfering RNA targeting hepatic PCSK9, has demonstrated efficacy in lowering LDL cholesterol across various adult populations; however, its efficacy and safety in paediatric patients with heterozygous familial hypercholesterolaemia (HeFH), a genetic disorder characterised by elevated LDL cholesterol levels and increased cardiovascular risk, have not been well established. To address this gap, the ORION-16 study evaluated the efficacy and safety of inclisiran in reducing LDL cholesterol levels in 141 adolescents with HeFH.
The study found that inclisiran treatment resulted in significantly greater reductions in LDL cholesterol from baseline to day 330 compared with placebo (-27.1% vs 1.4%), with a between-group difference of -28.5% in adolescents with HeFH. Sustained LDL cholesterol lowering was observed through day 720 in Part 2, with a mean percentage reduction of…
