Plozasiran for Managing Persistent Chylomicronemia and Pancreatitis Risk
Abstract
Background Persistent chylomicronemia is a genetic recessive disorder that is classically caused by familial chylomicronemia syndrome (FCS), but it also has multifactorial causes. The disorder is associated with the risk of recurrent acute pancreatitis. Plozasiran is a small interfering RNA that reduces hepatic production of apolipoprotein C-III and circulating triglycerides. Methods In a phase 3 trial, we randomly assigned 75 patients with persistent chylomicronemia (with or without a genetic diagnosis) to receive subcutaneous plozasiran (25 mg or 50 mg) or placebo every 3 months for 12 months. The primary end point was the median percent change from baseline in the fasting triglyceride level at 10 months. Key secondary end points were the percent change in the fasting triglyceride level from baseline to the mean of values at 10 months and 12 months, changes in the fasting apolipoprotein C-III level from baseline to 10 months and 12 months, and the incidence of acute pancreatitis. Results At baseline, the median triglyceride level was 2044 mg per deciliter. At 10 months, the median change from baseline in the fasting triglyceride level (the primary end point) was -80% in the 25-mg plozasiran group, -78% in the 50-mg plozasiran group, and -17% in the placebo group (P Conclusions Patients with persistent chylomicronemia who received plozasiran had significantly lower triglyceride levels and a lower incidence of pancreatitis than those who received placebo. (Funded by Arrowhead…
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In the phase 3 PALISADE trial, plozasiran reduced apoC-III by ~90% and TG by 78-80% at 10 months, achieving TG levels <500 mg/dL in about half of patients and <150 mg/dL in over 90% of those with partial LPL activity.
“In the phase 3 PALISADE trial [118], plozasiran reduced apoC-III by ~ 90% and TG by 78–80% at 10 months, achieving TG levels < 500 mg/dL in about half of patients and < 150 mg/dL in over 90% of those with partial LPL activity.”
From Targeting triglycerides for cardiovascular risk reduction (2026) · cites this paper as [118]
Four randomized controlled trials met eligibility criteria and were included in the meta-analysis.
“Ultimately, four RCTs met al.l eligibility criteria and were included in the final analysis [16, 17, 22, 23].”
From The Efficacy and Safety of Plozasiran on Lipid Profile in Dyslipidemic Disorders: A Systematic Review and Meta-Analysis (2026) · cites this paper as [17]
The meta-analysis included 1,514 participants, with 534 assigned to plozasiran and 980 receiving placebo.
“The meta-analysis included data from 4 clinical trials [16, 17, 22, 23], encompassing a total of 1,514 participants—534 assigned to various doses and treatment durations of plozasiran, and 980 receiving placebo.”
From The Efficacy and Safety of Plozasiran on Lipid Profile in Dyslipidemic Disorders: A Systematic Review and Meta-Analysis (2026) · cites this paper as [17]
Recent trials by Watts et al. (2025) and Gaudet et al. (2024) demonstrated that plozasiran achieved guideline-recommended triglyceride targets (<500 mg/dL) and reduced the incidence of acute pancreatitis events.
“In contrast, recent trials by Watts et al. (2025) [17] and Gaudet et al. (2024) [23] demonstrated that plozasiran was able to achieve guideline-recommended triglyceride targets (< 500 mg/dL) and reduce the incidence of acute pancreatitis events [Watts 2025; Gaudet 2024].”
From The Efficacy and Safety of Plozasiran on Lipid Profile in Dyslipidemic Disorders: A Systematic Review and Meta-Analysis (2026) · cites this paper as [17]
Plozasiran is associated with a higher incidence of headaches.
“Concerning safety findings for plozasiran, we observed slight increases in HbA1c, AST, and ALT levels, along with a higher incidence of headaches.”
From The Efficacy and Safety of Plozasiran on Lipid Profile in Dyslipidemic Disorders: A Systematic Review and Meta-Analysis (2026) · cites this paper as [17]
Watts et al. (2025) focused on individuals with genetically confirmed familial chylomicronemia syndrome (FCS) or symptomatic persistent chylomicronemia, aiming to assess reductions in acute pancreatitis events.
“Watts et al. (2025) focused on individuals with genetically confirmed familial chylomicronemia syndrome (FCS) or symptomatic persistent chylomicronemia, aiming to assess reductions in acute pancreatitis events [17].”
From The Efficacy and Safety of Plozasiran on Lipid Profile in Dyslipidemic Disorders: A Systematic Review and Meta-Analysis (2026) · cites this paper as [17]
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Atherogenic dyslipidaemia and residual cardiovascular risk: Understanding the link to heart disease
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New Approaches to Lipoproteins for the Prevention of Cardiovascular Events
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InfluentialGenetics of remnant cholesterol
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ApoC-III as Therapeutic Target: Is it Primetime for Clinical Use?
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“Recently, the preliminary findings of the PALISADE study [37], a Phase 3 placebo-controlled trial in adults with genetically confirmed or clinically diagnosed FCS, showed that subcutaneous plozasiran (25 mg or 50 mg) every three months for one year was able to reduce TG levels of 80% and 78% in the…”
Diretriz Brasileira de Dislipidemias e Prevenção da Aterosclerose – 2025
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Approach to the Adult Patient with Chylomicronemia
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“In a recent phase 3 trial, it was also found that the triglyceride levels of patients with persistent chylomicrons treated with plo-zasiran were significantly lower than those who received a placebo, and they had fewer adverse reactions (76).”
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InfluentialLi, D., Liu, Z., & Jiang, H. (2026) · Lipids in Health and Disease · doi:10.1186/s12944-026-02862-0 · Cited by 8 · see citing works
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Genetic determinants of pancreatitis risk in hypertriglyceridemia
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“Serious and severe adverse events occurred more in the placebo group [5].”
InfluentialEmerging agents targeting triglycerides
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“Such research will be essential to establish plozasiran’s role in routine clinical practice. familial chylomicronemia syndrome (FCS) or symptomatic persistent chylomicronemia, aiming to assess reductions in acute pancreatitis events [17].”
InfluentialOusey, J. A., Jomphe, C., Ta, A., Gosselin, N. H., & Shi, J. G. (2025) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.70055 · Cited by 5 · see citing works
“…3 trial conducted in FCS patients, plozasiran 25 and 50 mg Q3M dose regimens showed comparable PD activity, clinical efficacy, and safety profiles, 8 suggesting that the 25 mg Q3M dose will likely be safe and effective in adolescent FCS patients, in spite of the projected moderate increase in…”
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Pharmacotherapy of cardiovascular diseases from herbs and pills to nucleic acids
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“NCT05089084, “plozasiran for managing persistent chy-lomicronemia and pancreatitis risk” (PALISADE), is a phase 3, placebo-controlled trial published in 2024 that studied the effect of 25 or 50 mg of plozasiran quarterly for 12 months in patients with persistent chylomicronemia [49].”
Nucleic Acid–Based Therapies — The Next Frontier in Treating Dyslipidemias
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Self-citationAdvancing Gene Therapy for Pancreatitis: From Genetic Insights to Clinical Translation
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“The phase III results demonstrated that plozasiran treatment resulted in a 5-fold greater median TG reduction and lower AP incidence versus placebo [ 77 ].”
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Self-citationTherapeutic siRNA: Mechanisms, challenges, strategies, and clinical translation
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New and Emerging Therapeutic Targets for ApoB-Containing Particles Lowering
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“Lipid-lowering therapies have been shown to reduce the risk of RAP in patients with severe hypertriglyceridemia [28,29].”
Plozasiran for Managing Persistent Chylomicronemia and Pancreatitis Risk
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Self-citationSmall interfering RNA (siRNA) in dyslipidemia: a systematic review on safety and efficacy of siRNA
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Diagnosis, Evaluation, and Management of Severe Hypertriglyceridemia
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Adjunctive pharmacological strategies for residual risk reduction after myocardial revascularisation
Oshima, A., Serruys, P. W. J. C., Garg, S., McEvoy, J. W., Wood, D. A., Doenst, T., Taggart, D. P., Puskas, J. D., Shajahan, A., Sharif, F., et al. (2026) · EuroIntervention · doi:10.4244/eij-d-25-00598 · Cited by 3 · see citing works
Ballantyne, C. M., Baass, A. W., Rosenson, R. S., Hegele, R. A., Nicholls, S. J., Vasas, S., Clifton, P. M., Lucas, K. J., Páll, D., Zhou, R., et al. (2026) · American Journal of Preventive Cardiology · doi:10.1016/j.ajpc.2026.101523 · Cited by 3 · see citing works
“…from severe dietary fat restriction, which is supported mainly by observational and case-series data, there are currently no effective therapeutic options proven to reduce the risk of AP in patients with hypertriglyceridemia outside of patients with Familial Chylomicronemia Syndrome (FCS) [8 – 12].”
Self-citationGinsberg, H. N., & Bornfeldt, K. (2026) · Circulation Research · doi:10.1161/circresaha.125.327267 · Cited by 4 · see citing works
“…APOC3 silencing, on the other hand, reduced both TRL and LDL particle concentrations in this mouse model, 85 and importantly, reduced TRL levels in patients with homozygous loss of function of LPL. 135,137 The latter results support increased TRL uptake by the liver when APOC3 levels are reduced.”
Reducing the Risk of Pancreatitis by Inhibiting APOC3
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“During disease progression, AP can lead to systemic inflammation and multiorgan dysfunction, and in advanced stages, sepsis-related complications may occur, which is a significant cause of mortality in these patients (Watts et al. 2025 ).”
D’Erasmo, L., Tramontano, D., & Arca, M. (2025) · Current Opinion in Endocrinology Diabetes and Obesity · doi:10.1097/med.0000000000000941 · Cited by 2 · see citing works
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Zadeh, S. S. T., Toth, P. P., & Banach, M. (2026) · Frontiers in Pharmacology · doi:10.3389/fphar.2026.1789019 · Cited by 2 · see citing works
“Treatment was also associated with an 83% reduction in the risk of acute pancreatitis compared with placebo (Watts et al., 2025).”
ApoC[III] You Later: A Review of Plozasiran for the Management of Familial Chylomicronemia Syndrome
Yin, E. B., Wanat, M. A., & Wang, E. M. (2026) · Annals of Pharmacotherapy · doi:10.1177/10600280261452675 · Cited by 2 · see citing works
Design of Nanocarriers for Kidney Targeted Delivery of Nucleic Acid Therapeutics
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“To date, eight siRNA therapeutics (patisiran, givosiran, lumasiran, inclisiran, vutrisiran, nedosiran, fitusiran, and plozasiran) have been approved for clinical use, and more than 35 candidates are currently in clinical development [ 73–75 ].”
FNIP1 variants are associated with favourable metabolism in 1 million humans
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Molecular Therapeutics in Development to Treat Hyperlipoproteinemia
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“PALISADE was a multicenter, double-blind, placebo-controlled, phase III trial with 75 participants with persistent chylomicronemia due to genetically or clinically defined FCS despite stable standard-of-care treatment [72].”
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“No patients experienced alanine aminotransferase or aspartate aminotransferase levels > 3 × the upper limit of normal, and platelet levels remained similar to baseline in all groups [18].”
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Elbahloul, M. A., Gamal, A., Maihoub, O., Elbaz Hassan, M. M., Attia, M. K., Salah, A. M., Elboraay, T., Awad, A. K., Akoum, A., Elazab, A., et al. (2026) · Diabetes, Obesity and Metabolism · doi:10.1111/dom.70717 · Cited by 1 · see citing works
“Only 3 studies reported the incidence of pancreatitis [7, 42, 44].”
Novel agents for treating severe hypertriglyceridemia
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Pediatric Hypertriglyceridemia: Lipoprotein Metabolism, Etiology, and Management
Moriyama, H., & Ham, J. N. (2025) · Current Pediatrics Reports · doi:10.1007/s40124-025-00349-7 · Cited by 1 · see citing works
“The ploza-siran studies underway include trials in patients with FCS (the PALISDADE study), patients with non-FCS related severe hypertriglyceridemia (SHASTA), and patients with mixed hyperlipidemia (MUIR) [72–74].”
- Authors
- Watts, G. F., Rosenson, R. S., Hegele, R. A., Goldberg, I. J., Gallo, A., Mertens, A., Baass, A., Zhou, R., Muhsin, M., Hellawell, J., Leeper, N. J., & Gaudet, D.
- Affiliations
- Inserm (FR)
- ORCID
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2025-01-09
- Volume
- 392
- Issue
- 2
- Pages
- 127-137
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- OpenAlex
- W4402146754
- Semantic Scholar
- 7e77ad0e7b828bad027c2dd2d373cdb99622ce79
- Cited by
- 178
- References
- 42
- Citations by year
- 2026: 82 · 2025: 81 · 2024: 15
- FWCI
- 47.48
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- New England Journal of Medicine (publishedVersion)
- PubMed (publishedVersion)
- UWA Profiles and Research Repository (UWA) (submittedVersion)
- Lirias (KU Leuven) (submittedVersion)
- Funders
- Arrowhead Pharmaceuticals
- Topics
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Lipid metabolism and disorders
- Health Sciences › Medicine › Surgery › Lipoproteins and Cardiovascular Health
- Health Sciences › Medicine › Surgery › Lymphatic Disorders and Treatments
- Keywords
- plozasiran, familial chylomicronemia syndrome, acute pancreatitis, apolipoprotein C-III, triglycerides, siRNA, hypertriglyceridemia, hyperglycemia, adverse events, prediabetes, diabetes mellitus, familial hypertriglyceridemia, familial combined hyperlipidemia
- MeSH terms
- Acute Disease, Adult, Aged, Double-Blind Method, Female, Humans, Injections, Subcutaneous, Hyperlipoproteinemia Type I, Male, Middle Aged, Pancreatitis, Triglycerides, Incidence, RNA, Small Interfering, Apolipoprotein C-III, Young Adult
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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