Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia

Summary

This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).

Timeline

Start

2021-01-27

Primary completion

2023-11-09

Completion

2024-11-27

Outcome

Met primary endpoint

registry analysis (superiority test); Part 1- Inclisiran vs Part 1 - Placebo; p <.0001; LS Mean -28.54 (95% CI -35.81 to -21.27); ANCOVA NCT04652726 ↗

Publications

• Wiegman A, Peterson AL, Bruckert E, Defesche JC, Schweizer A, Bergeron J, Chueca MJ, Roeters van Lennep JE, Santos RD, Schwab KO, Lesogor A, Zhang S, Hegele RA. Efficacy and safety of inclisiran in adolescents with heterozygous familial hypercholesterolaemia (ORION-16): a two-part, randomised, multicentre clinical trial. Lancet Diabetes Endocrinol. 2026 Mar;14(3):233-242. doi: 10.1016/S2213-8587(25)00351-1. Epub 2026 Jan 27.
• Reijman MD, Schweizer A, Peterson ALH, Bruckert E, Stratz C, Defesche JC, Hegele RA, Wiegman A. Rationale and design of two trials assessing the efficacy, safety, and tolerability of inclisiran in adolescents with homozygous and heterozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2022 Jul 20;29(9):1361-1368. doi: 10.1093/eurjpc/zwac025.
• Warden BA, Duell PB. Inclisiran: A Novel Agent for Lowering Apolipoprotein B-containing Lipoproteins. J Cardiovasc Pharmacol. 2021 Aug 1;78(2):e157-e174. doi: 10.1097/FJC.0000000000001053.

Drugs

Indications