A weekly digest of FCS and severe high-triglyceride research, treatments, regulation and devices.

每周汇总 FCS 与重度高甘油三酯的研究、新药、监管与仪器进展。

FCS Weekly — Issue 1 · November 13, 2026

Qingyang · FCS Foundation Board Member & Tech Chair, FCS Foundation

This week's top stories

Plozasiran cuts pancreatitis risk by 78% in severe hypertriglyceridemia — full SHASTA-3/4 results published in the New England Journal of Medicine.
Three years on: the PALISADE extension shows plozasiran's triglyceride lowering holds up in FCS.
A first for gene editing: one infusion of CTX310 cut triglycerides 63% in a small early trial — while Ionis halves its 2026 Tryngolza sales outlook.

Research & new treatments

Plozasiran for severe hypertriglyceridemia: pancreatitis risk down 78% (NEJM, Nov 5)

Two identical Phase 3 trials (SHASTA-3 and SHASTA-4) enrolled 757 adults with severe hypertriglyceridemia, randomized 2:1 to plozasiran 25 mg or placebo every three months. At 12 months, median triglycerides were down 79% and 81% from baseline in the two trials; 91% and 93% of patients on plozasiran reached triglycerides below 500 mg/dL, versus about half on placebo. Adjudicated acute pancreatitis occurred at 1 vs 5 events per 100 patient-years (rate ratio 0.22, P = 0.008) — a 78% lower rate. Adverse events occurred in 73% of patients in both arms. The trials were funded by Arrowhead Pharmaceuticals. Note this was in severe hypertriglyceridemia broadly, not FCS specifically.

Three-year PALISADE data in FCS (AHA Scientific Sessions, Nov 7–9)

In the PALISADE long-term extension, 65 of the original 75 participants continued treatment; median baseline triglycerides were 2,259 mg/dL. Through 24 months of the extension — up to 36 months of total treatment — median triglycerides stayed 76% below baseline and APOC3 89% below; 57% of patients were below 880 mg/dL and 48% below 500 mg/dL at month 24. Three adjudicated pancreatitis events were reported. The extension had no placebo group, so these results are descriptive. Presented by Dr. Robert Rosenson; Arrowhead press release, Nov 7.

One-shot gene editing for severe high triglycerides: CTX310 (AHA, Nov 9)

CRISPR Therapeutics' CTX310 — a one-time, in vivo CRISPR/Cas9 therapy that edits the ANGPTL3 gene in the liver — reported first Phase 1b data in 10 patients with severe hypertriglyceridemia (median baseline triglycerides above 1,000 mg/dL). A single infusion cut ANGPTL3 by a mean 65% and triglycerides by 63% at day 30, with reductions maintained at 90 days in the seven patients followed that long. There were no treatment-related serious adverse events and no clinically relevant liver enzyme elevations. The FDA has granted CTX310 Regenerative Medicine Advanced Therapy (RMAT) designations for severe hypertriglyceridemia and elevated LDL cholesterol. Caveats: tiny, open-label, short follow-up, company-reported and not yet peer-reviewed — early promise, not proof.

Blood purification for triglyceride-induced pancreatitis: what helps, and when (Nov 8)

A single-center retrospective study of 204 patients with hypertriglyceridemia-induced acute pancreatitis compared hemoperfusion plus continuous venovenous hemofiltration (HP + CVVH) with CVVH alone. Overall, adding hemoperfusion did not reduce severe pancreatitis — but in patients whose CRP (an inflammation marker) was roughly 195–297 mg/L, it did. Secondary measures (inflammation levels, ICU stay, time to feeding) looked better with the combination, at higher blood-purification costs. Takeaway: any benefit depends on how inflamed the patient is — a decision for the ICU team, not a routine add-on.

Recently published — not this week's news

A rare cause of FCS in a 5-month-old baby (JCEM Case Reports, published Oct 5, 2026): FCS caused by a homozygous variant in the LMF1 gene — an exceedingly rare cause, and presentation in infancy is rarer still. Early diagnosis and a low-fat diet brought triglycerides down and prevented pancreatitis. A reminder that FCS can appear in infancy, and that genetic testing can find the cause.
Plasma exchange in severe hypertriglyceridemia emergencies (Molecular Genetics and Metabolism Reports, Sept 2026): a 21-patient retrospective case series from a multinational group found therapeutic plasma exchange and/or exchange transfusion rapidly lowered lipids and eased symptoms in emergency admissions, including recurrent pancreatitis cases. Retrospective and uncontrolled — evidence of use, not of superiority over standard care.

Regulation & access

England and Wales: NICE backs both olezarsen and plozasiran for FCS (Oct 28, via Action FCS): NICE accepted both medicines for use in England and Wales under its cost-comparison process — the first time people with FCS there have a choice of primary treatment alongside diet. Action FCS and Metabolic Support UK contributed patient evidence to the appraisal.
Europe: CHMP positive opinion for Tryngolza in severe hypertriglyceridemia (Oct 16; noted in Ionis's Q3 results, Nov 9): EMA's committee recommended extending Tryngolza (olezarsen) to adults with severe hypertriglyceridemia (fasting triglycerides ≥880 mg/dL); a final European Commission decision is expected in Q4 2026. If confirmed, it would be the first RNA-targeted therapy authorized in the EU for severe hypertriglyceridemia at that level.

Industry

Ionis Q3: Tryngolza sales ramp slower than expected (company press release, Nov 9): TRYNGOLZA U.S. net sales were $9M in Q3 ($41M over nine months); Ionis cut its 2026 Tryngolza sales guidance to about $50M, from $100–110M, citing a more gradual launch ramp. Total revenue guidance ($875–900M) was reaffirmed. For patients, the practical read: the drug is available, but uptake is slow — access and awareness remain the bottleneck, not approval.

Community

FCS Awareness Day, November 6: The Foundation for Triglyceride Disorders marked FCS Awareness Day with patient and caregiver voices, and Action FCS ran its 10g Fat Challenge — a day on no more than 10g of fat — with 10 days of low-fat recipes to mark its 10th anniversary.

Join us

Living with FCS or caring for someone who is? Join the FCS Support Group on Facebook: facebook.com/groups/livingwithfcs — patients, families and caregivers sharing practical experience.

Sources

Watts GF et al. Plozasiran for Severe Hypertriglyceridemia and Risk of Pancreatitis. N Engl J Med. 2026;395(18):1805–1817. https://pubmed.ncbi.nlm.nih.gov/42981569/
Arrowhead Pharmaceuticals. Arrowhead Pharmaceuticals Presents Plozasiran Data Highlighting Sustained Triglyceride Reductions and Reduced Acute Pancreatitis Rates (press release, Nov 7, 2026). https://www.businesswire.com/news/home/20261107972891/en/Arrowhead-Pharmaceuticals-Presents-Plozasiran-Data-Highlighting-Sustained-Triglyceride-Reductions-and-Reduced-Acute-Pancreatitis-Rates
HCPLive. PALISADE Extension Supports Plozasiran's Efficacy, Safety in Familial Chylomicronemia (Nov 9, 2026). https://www.hcplive.com/view/palisade-extension-supports-plozasiran-s-efficacy-safety-in-familial-chylomicronemia
CRISPR Therapeutics. CRISPR Therapeutics Presents Phase 1b Data for CTX310 Demonstrating Reductions in ANGPTL3 and Triglycerides in Patients with Severe Hypertriglyceridemia at the American Heart Association Scientific Sessions 2026 (press release, Nov 9, 2026). https://investingnews.com/crispr-therapeutics-presents-phase-1b-data-for-ctx310-r-demonstrating-reductions-in-angptl3-and-triglycerides-in-patients-with-severe-hypertriglyceridemia-at-the-american-heart-association-scientific-/
Ionis Pharmaceuticals. Ionis reports third quarter 2026 financial results and highlights progress on key programs (press release, Nov 9, 2026). https://www.stockwatch.com/News/Item/U-b20261109740265-U!IONS-20261109/U/IONS
Li A et al. Efficacy Heterogeneity of Hemoperfusion Plus Continuous Venovenous Hemofiltration for Hypertriglyceridemia-Induced Acute Pancreatitis Stratified by Severity of Inflammatory Response. Ther Apher Dial. 2026 Nov 8 (online ahead of print). https://pubmed.ncbi.nlm.nih.gov/43000034/
Kumar PR et al. A rare case of familial chylomicronemia in infancy due to homozygous LMF1 variant. JCEM Case Rep. 2026;4(11):luag164. https://pubmed.ncbi.nlm.nih.gov/42831121/
Stefanutti C et al. Efficacy of plasma exchange and exchange transfusion in the clinical management of severe hypertriglyceridemia in emergency. Mol Genet Metab Rep. 2026;12:101353. https://pubmed.ncbi.nlm.nih.gov/42756739/
Action FCS. NICE publishes guidelines for new therapies (Oct 28, 2026). https://www.actionfcs.org/news/nice-publishes-guidelines-for-new-therapies/
Sobi. Sobi receives positive CHMP opinion for TRYNGOLZA (olezarsen) for the treatment of severe hypertriglyceridemia (sHTG) (Oct 16, 2026). https://mb.cision.com/Main/14266/4409832/4321959.pdf
The Foundation for Triglyceride Disorders. The Foundation for Triglyceride Disorders Marks FCS Awareness Day, Nov. 6, With the Voices of Patients and Caregivers (press release, Nov 5, 2026). https://www.morningstar.com/news/pr-newswire/20261105la90497/the-foundation-for-triglyceride-disorders-marks-fcs-awareness-day-nov-6-with-the-voices-of-patients-and-caregivers
Action FCS. FCS Awareness Day 2026. https://www.actionfcs.org/get-involved/events/fcs-awareness-day-2026/

Company press releases are marked as such; their figures have not been peer-reviewed. This digest summarizes published research and company announcements for information only. It is not medical advice — talk to your doctor before making any change to your treatment or diet.

FCS 一周 · 第 1 期 · 2026 年 11 月 13 日

清阳 · FCS Foundation Board Member & Tech Chair, FCS Foundation（基金会理事、技术负责人；本人也是 FCS 患者）

本周要点

Plozasiran 让重度高甘油三酯患者的胰腺炎风险下降 78%——SHASTA-3/4 完整结果发表于《新英格兰医学杂志》。
三年随访：PALISADE 延长研究显示，plozasiran 在 FCS 患者中的降甘油三酯效果持续保持。
基因编辑首秀：一次输注的 CTX310 在早期试验中降甘油三酯 63%；与此同时，Ionis 把 Tryngolza 全年销售预期砍掉一半。

研究与新药

Plozasiran 治疗重度高甘油三酯：胰腺炎风险下降 78%（《新英格兰医学杂志》，11 月 5 日）

两项设计相同的 3 期试验（SHASTA-3 与 SHASTA-4）共纳入 757 名重度高甘油三酯成人，按 2:1 随机接受 plozasiran 25 mg 或安慰剂，每三个月注射一次。12 个月时，两项试验中位甘油三酯较基线分别下降 79% 和 81%；plozasiran 组 91% 和 93% 的患者甘油三酯降到 500 mg/dL 以下，安慰剂组只有约一半。经判定的急性胰腺炎发生率为每 100 人年 1 例 vs 5 例（率比 0.22，P = 0.008），即下降 78%。不良事件发生率两组均为 73%。试验由 Arrowhead Pharmaceuticals 资助。注意：这是针对重度高甘油三酯人群（不只 FCS）的研究。

FCS 的三年 PALISADE 数据（美国心脏协会年会，11 月 7–9 日）

PALISADE 长期延长研究中，原 75 名受试者有 65 人继续用药，中位基线甘油三酯为 2,259 mg/dL。延长 24 个月时（总用药最长 36 个月），中位甘油三酯仍比基线低 76%，APOC3 低 89%；第 24 个月时 57% 的患者甘油三酯低于 880 mg/dL，48% 低于 500 mg/dL。期间共报告 3 起经判定的胰腺炎事件。延长研究没有安慰剂对照组，因此结果为描述性。由 Robert Rosenson 医生报告；Arrowhead 新闻稿，11 月 7 日。

一次给药的基因编辑疗法 CTX310（美国心脏协会年会，11 月 9 日）

CRISPR Therapeutics 的 CTX310 是一种一次性体内 CRISPR/Cas9 疗法，通过编辑肝脏中的 ANGPTL3 基因起效。首批 1b 期数据显示，10 名重度高甘油三酯患者（中位基线甘油三酯超过 1,000 mg/dL）接受单次静脉输注后，第 30 天 ANGPTL3 平均下降 65%，甘油三酯下降 63%；随访满 90 天的 7 名患者效果持续。未出现与治疗相关的严重不良事件，也无有临床意义的肝酶升高。FDA 已授予 CTX310 针对重度高甘油三酯和 LDL 胆固醇升高的再生医学先进疗法（RMAT）资格。提醒：样本很小、无对照、随访短，且为药企发布、未经同行评审——是早期希望，不是定论。

甘油三酯引起的胰腺炎：血液净化什么时候有用？（11 月 8 日）

一项单中心回顾性研究纳入 204 名高甘油三酯性急性胰腺炎患者，比较血液灌流联合连续静脉-静脉血液滤过（HP+CVVH）与单用 CVVH。总体上，加血液灌流并不能降低重症胰腺炎发生率——但在 CRP（炎症指标）约 195–297 mg/L 的患者中，它确实有效。联合治疗在炎症指标、ICU 住院时间、恢复进食时间等次要指标上更好看，但血液净化费用更高。结论：有没有获益取决于患者当时的炎症程度，这是 ICU 团队的个体化决定，不是常规加项。

最近发表——不是本周新出

5 个月大婴儿的罕见 FCS 病因（《JCEM 病例报告》，2026 年 10 月 5 日发表）：患儿因 LMF1 基因纯合变异导致 FCS——这是极罕见的病因，而婴儿期发病更为罕见。早期诊断加低脂饮食使甘油三酯明显下降，避免了胰腺炎。提醒：FCS 也可能在婴儿期出现，基因检测可以找到病因。
重度高甘油三酯急症中的血浆置换（《分子遗传与代谢报告》，2026 年 9 月）：一个多国团队的 21 例回顾性病例系列发现，治疗性血浆置换和/或换血输血能快速降低急诊入院患者的血脂、缓解症状，包括反复胰腺炎的病例。这是回顾性、无对照的研究——证明的是“可用”，不是“优于标准治疗”。

监管与可及性

英格兰和威尔士：NICE 同时批准 olezarsen 与 plozasiran 用于 FCS（10 月 28 日，来源：Action FCS）：NICE 通过成本比较流程接受两种药物在英格兰和威尔士使用——这是当地 FCS 患者第一次在饮食之外有了两种一线治疗选择。Action FCS 与 Metabolic Support UK 为评审提供了患者 lived experience 证据。
欧洲：CHMP 对 Tryngolza 用于重度高甘油三酯给出积极意见（10 月 16 日；Ionis 三季报中提及，11 月 9 日）：欧洲药品管理局人用药品委员会建议将 Tryngolza（olezarsen）适应症扩展至重度高甘油三酯成人（空腹甘油三酯 ≥880 mg/dL）；欧盟委员会最终决定预计在 2026 年第四季度作出。如获确认，这将是欧盟首个获批用于该人群的 RNA 靶向疗法。

行业动态

Ionis 三季报：Tryngolza 销售爬坡慢于预期（药企新闻稿，11 月 9 日）：TRYNGOLZA 美国三季度净销售额 900 万美元，前九个月 4,100 万美元；Ionis 把 2026 年 Tryngolza 销售指引从 1–1.1 亿美元下调至约 5,000 万美元，原因是上市爬坡比预期缓慢。全年总收入指引（8.75–9 亿美元）维持不变。对患者而言实际含义是：药已经上市了，但用上的人还不多——瓶颈在可及性和知晓度，不在审批。

社区

FCS 宣传日，11 月 6 日：The Foundation for Triglyceride Disorders（甘油三酯疾病基金会）以患者与照护者的声音纪念 FCS 宣传日；英国 Action FCS 开展了 10g 脂肪挑战——一天只吃不超过 10g 脂肪，并发布 10 天低脂食谱，庆祝机构成立 10 周年。

加入我们

如果你或家人是 FCS / 严重高甘油三酯患者，欢迎加入 FCS 患者家属微信群：加清阳微信 Qing-Yang_Li（请注明 FCS 入群），和病友、家属一起交流实用经验。

来源

Watts GF 等. Plozasiran for Severe Hypertriglyceridemia and Risk of Pancreatitis. N Engl J Med. 2026;395(18):1805–1817. https://pubmed.ncbi.nlm.nih.gov/42981569/
Arrowhead Pharmaceuticals. Arrowhead Pharmaceuticals Presents Plozasiran Data Highlighting Sustained Triglyceride Reductions and Reduced Acute Pancreatitis Rates（新闻稿，2026 年 11 月 7 日）. https://www.businesswire.com/news/home/20261107972891/en/Arrowhead-Pharmaceuticals-Presents-Plozasiran-Data-Highlighting-Sustained-Triglyceride-Reductions-and-Reduced-Acute-Pancreatitis-Rates
HCPLive. PALISADE Extension Supports Plozasiran's Efficacy, Safety in Familial Chylomicronemia（2026 年 11 月 9 日）. https://www.hcplive.com/view/palisade-extension-supports-plozasiran-s-efficacy-safety-in-familial-chylomicronemia
CRISPR Therapeutics. CRISPR Therapeutics Presents Phase 1b Data for CTX310 Demonstrating Reductions in ANGPTL3 and Triglycerides in Patients with Severe Hypertriglyceridemia at the American Heart Association Scientific Sessions 2026（新闻稿，2026 年 11 月 9 日）. https://investingnews.com/crispr-therapeutics-presents-phase-1b-data-for-ctx310-r-demonstrating-reductions-in-angptl3-and-triglycerides-in-patients-with-severe-hypertriglyceridemia-at-the-american-heart-association-scientific-/
Ionis Pharmaceuticals. Ionis reports third quarter 2026 financial results and highlights progress on key programs（新闻稿，2026 年 11 月 9 日）. https://www.stockwatch.com/News/Item/U-b20261109740265-U!IONS-20261109/U/IONS
Li A 等. Efficacy Heterogeneity of Hemoperfusion Plus Continuous Venovenous Hemofiltration for Hypertriglyceridemia-Induced Acute Pancreatitis Stratified by Severity of Inflammatory Response. Ther Apher Dial. 2026 年 11 月 8 日（在线优先发表）. https://pubmed.ncbi.nlm.nih.gov/43000034/
Kumar PR 等. A rare case of familial chylomicronemia in infancy due to homozygous LMF1 variant. JCEM Case Rep. 2026;4(11):luag164. https://pubmed.ncbi.nlm.nih.gov/42831121/
Stefanutti C 等. Efficacy of plasma exchange and exchange transfusion in the clinical management of severe hypertriglyceridemia in emergency. Mol Genet Metab Rep. 2026;12:101353. https://pubmed.ncbi.nlm.nih.gov/42756739/
Action FCS. NICE publishes guidelines for new therapies（2026 年 10 月 28 日）. https://www.actionfcs.org/news/nice-publishes-guidelines-for-new-therapies/
Sobi. Sobi receives positive CHMP opinion for TRYNGOLZA (olezarsen) for the treatment of severe hypertriglyceridemia (sHTG)（2026 年 10 月 16 日）. https://mb.cision.com/Main/14266/4409832/4321959.pdf
The Foundation for Triglyceride Disorders. The Foundation for Triglyceride Disorders Marks FCS Awareness Day, Nov. 6, With the Voices of Patients and Caregivers（新闻稿，2026 年 11 月 5 日）. https://www.morningstar.com/news/pr-newswire/20261105la90497/the-foundation-for-triglyceride-disorders-marks-fcs-awareness-day-nov-6-with-the-voices-of-patients-and-caregivers
Action FCS. FCS Awareness Day 2026. https://www.actionfcs.org/get-involved/events/fcs-awareness-day-2026/

药企新闻稿已在正文中标注，其数据未经同行评审。本周报仅为公开研究与药企公告的摘要整理，不构成医疗建议。用药或饮食调整请咨询医生。

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FCS Weekly — Issue 1 · November 13, 2026 2026-11-13