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Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia
Open accessAbstract
BACKGROUND: Familial hypercholesterolemia is characterized by an elevated level of low-density lipoprotein (LDL) cholesterol and an increased risk of premature atherosclerotic cardiovascular disease. Monoclonal antibodies directed against proprotein convertase subtilisin-kexin type 9 (PCSK9) have been shown to reduce LDL cholesterol levels by more than 50% but require administration every 2 to 4 weeks. In a phase 2 trial, a twice-yearly injection of inclisiran, a small interfering RNA, was shown to inhibit hepatic synthesis of PCSK9 in adults with heterozygous familial hypercholesterolemia. METHODS: In this phase 3, double-blind trial, we randomly assigned, in a 1:1 ratio, 482 adults who had heterozygous familial hypercholesterolemia to receive subcutaneous injections of inclisiran sodium (at a dose of 300 mg) or matching placebo on days 1, 90, 270, and 450. The two primary end points were the percent change from baseline in the LDL cholesterol level on day 510 and the time-adjusted percent change from baseline in the LDL cholesterol level between day 90 and day 540. RESULTS: The median age of the patients was 56 years, and 47% were men; the mean baseline level of LDL cholesterol was 153 mg per deciliter. At day 510, the percent change in the LDL cholesterol level was a reduction of 39.7% (95% confidence interval [CI], -43.7 to -35.7) in the inclisiran group and an increase of 8.2% (95% CI, 4.3 to 12.2) in the placebo group, for a between-group difference of -47.9 percentage points (95% CI, -53.5 to -42.3; P<0.001). The time-averaged percent change in the LDL cholesterol level between day 90 and day 540 was a reduction of 38.1% (95% CI, -41.1 to -35.1) in the inclisiran group and an increase of 6.2% (95% CI, 3.3 to 9.2) in the placebo group, for a between-group difference of -44.3 percentage points (95% CI, -48.5 to -40.1; P<0.001). There were robust reductions in LDL cholesterol levels in all genotypes of familial hypercholesterolemia. Adverse events and serious adverse events were similar in the two groups. CONCLUSIONS: Among adults with heterozygous familial hypercholesterolemia, those who received inclisiran had significantly lower levels of LDL cholesterol than those who received placebo, with an infrequent dosing regimen and an acceptable safety profile. (Funded by the Medicines Company; ORION-9 ClinicalTrials.gov number, NCT03397121.).
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“Inclisiran lowered LDL-C by 47.9% in the 482 HeFH patients enrolled in ORION-9 at Day 510 [39].”
From Inclisiran SiRNA therapy for durable LDL-C reduction: a systematic review and meta-analysis highlighting a breakthrough in long-term cardiovascular risk management (2026) · cites this paper as [39]
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“The analysis of 11 RCTs for quantitative synthesis included more than 13,000 participants showed inclisiran produces significant LDL-C reduction versus placebo treatment [24, 25, 38–44, 46, 49].”
From Inclisiran SiRNA therapy for durable LDL-C reduction: a systematic review and meta-analysis highlighting a breakthrough in long-term cardiovascular risk management (2026) · cites this paper as [39]
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“The study by Raal et al. focused on HeFH patients displayed an outlier position in the lower left quadrant of the funnel because it had both a small effect size and a wide standard error [39].”
From Inclisiran SiRNA therapy for durable LDL-C reduction: a systematic review and meta-analysis highlighting a breakthrough in long-term cardiovascular risk management (2026) · cites this paper as [39]
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“These results were confirmed in the phase 3 ORION-9 trial which showed a − 48% LDL-C reduction of inclisiran relative to placebo among 482 patients with HeFH [138].”
From Established and Emerging Lipid-Lowering Drugs for Primary and Secondary Cardiovascular Prevention (2023) · cites this paper as [138]
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“Inclisiran improved multiple lipid parameters including non-HDL-C, apoB, triglycerides and lipoprotein(a) in addition to LDL-C which makes it beneficial for complete lipid management [39, 43, 49].”
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Metadata
- Authors
- Raal, F. J., Kallend, D., Ray, K. K., Turner, T., Koenig, W., Wright, R. S., Wijngaard, P. L. J., Curcio, D., Jaros, M. J., Leiter, L. A., & Kastelein, J. J. P.
- Affiliations
- University of the Witwatersrand (ZA)
- Medpace (United States) (US)
- From the Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa (F.J.R.); the Medicines Company, Zurich, Switzerland (D.K.); the Imperial Centre for Cardiovascular Disease Prevention, Department of Primary Care and Public Health, Imperial College, London (K.K.R.); Medpace Reference Laboratories, Cincinnati (T.T.); Deutsches Herzzentrum München, Technische Universität München, and German Center for Cardiovascular Research, Munich Heart Alliance, Munich (W.K.), and the...
- Mayo Clinic (US)
- St. Michael's Hospital (CA)
- ORCID
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2020-04-16
- Volume
- 382
- Issue
- 16
- Pages
- 1520-1530
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- OpenAlex
- W3011005820
- Semantic Scholar
- cd7f15bcb062953bf710c34645a08f7b4818c4d9
- Cited by
- 818
- References
- 52
- Citations by year
- 2026: 88 · 2025: 126 · 2024: 142 · 2023: 154 · 2022: 140 · 2021: 111 · 2020: 56
- FWCI
- 105.56
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- New England Journal of Medicine (publishedVersion)
- PubMed (publishedVersion)
- Pure Amsterdam UMC (publishedVersion)
- ENLIGHTEN (Jurnal Bimbingan dan Konseling Islam) (acceptedVersion, cc-by-sa)
- Data Archiving and Networked Services (DANS) (submittedVersion)
- mediaTUM (Technical University of Munich) (submittedVersion)
- Spiral (Imperial College London) (submittedVersion)
- Topics
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Lipoproteins and Cardiovascular Health
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Lipid metabolism and disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Biochemistry › Cholesterol and Lipid Metabolism
- Keywords
- inclisiran, heterozygous familial hypercholesterolemia, PCSK9 inhibition, siRNA, LDL cholesterol lowering, atherosclerotic cardiovascular disease prevention, hypercholesterolemia treatment, lipid-lowering therapy, PCSK9 monoclonal antibodies
- MeSH terms
- Proprotein Convertase 9, PCSK9 Inhibitors, Adult, Anticholesteremic Agents, Double-Blind Method, Female, Humans, Hyperlipoproteinemia Type II, Injections, Subcutaneous, Cholesterol, LDL, Male, Middle Aged, Treatment Outcome, RNA, Small Interfering
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