Amvuttra (vutrisiran) / Alnylam, Sanofi, Royalty - LARVOL DELTA

When Colon Biopsies Mislead: Refractory Diarrhea as a Manifestation of GI ATTRv Amyloidosis After Heart and Liver Transplantation (ACG 2026) - "Case Description/ A 71-year-old male with hereditary ATTR cardiac amyloidosis, status post liver and heart transplant (2016), on vutrisiran, presented with chronic refractory diarrhea...He failed loperamide, diphenoxylate, tincture of opium, codeine, antibiotics, cholestyramine, and fiber...Third, diarrhea in GI amyloidosis is multifactorial-including SIBO, malabsorption, protein-losing enteropathy, rapid transit, and autonomic neuropathy-and remains difficult to manage even after surgery. Figure: Figure 1 and 2 Figure: Figure 3"

Biopsy • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • CNS Disorders • Gastroenterology • Gastrointestinal Disorder • Immunology • Transplantation

Multimodality Imaging Reveals Rare Hereditary Transthyretin Amyloidosis Mimicking Hypertrophic Cardiomyopathy. (PubMed, JACC Case Rep) - "Unexplained hypertrophy with systemic manifestations should prompt evaluation for transthyretin amyloidosis."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Hypertrophic Cardiomyopathy • Non-obstructive Hypertrophic Cardiomyopathy • Pain

Specific treatment of ATTR-CM in Germany-expert consensus of the German Society of Amyloid Diseases (DGAK) and German Cardiac Society (DGK). (PubMed, Clin Res Cardiol) - "This consensus provides a practical framework for the specific treatment of ATTR-CM in Germany, aiming for early treatment initiation. Local standards and shared decision-making remain essential for the establishment of ATTR-CM therapy in an individual patient."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Molecular imaging of TTR amyloidosis: New data. (PubMed, Hell J Nucl Med) - "This narrative review synthesizes recent data on molecular imaging, emphasizing the central role of nuclear medicine physicians in applying multimodality diagnostic algorithms, recognizing typical and equivocal scan patterns. It further explores emerging roles for quantitative SPECT/CT and amyloid PET tracers in monitoring response to disease modifying therapies, including tetramer stabilizers (tafamidis, acoramidis), RNA interference silencers (vutrisiran, patisiran), and investigational fibril targeting antibodies and gene editing strategies, underscoring a shift from purely diagnostic imaging toward dynamic treatment monitoring in ATTR CM."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Congestive Heart Failure • Gastrointestinal Disorder • Heart Failure

Characteristics And Treatment Patterns In Patients With ATTR-CM Treated With Vutrisiran In Clinical Practice [Poster no. 716] (HFSA 2026) - No abstract available

Clinical • Cardiovascular

Long-Term Vutrisiran Reduces Cardiovascular Events And Hospitalizations In Patients With Transthyretin Cardiac Amyloidosis In The HELIOS-B One-Year Open Label Extension (HFSA 2026) - "Patients were assessed as the overall population (all patients), the monotherapy population (patients not on tafamidis at baseline; tafamidis drop-in therapy was allowed at any time) and the baseline tafamidis subgroup (patients receiving tafamidis at baseline). At rollover into OLE, patients in the monotherapy population receiving placebo during the DB (placebo-to-vutrisiran) had more advanced disease than those initially randomized to vutrisiran (long-term vutrisiran) or those in the baseline tafamidis subgroup (Table). These data demonstrate that long-term vutrisiran continues to reduce the risk of CV events and hospitalizations relative to patients originally randomized to placebo. Reduction in non-CV hospitalizations may suggest a systemic effect of therapy. Many treatment effects at 48M were larger than those previously reported at 36M, highlighting the benefits of early diagnosis and treatment."

Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Comparative Outcomes Of Tafamidis Plus Vutrisiran Versus Tafamidis Monotherapy In Transthyretin Amyloid Cardiomyopathy [Poster no. 570] (HFSA 2026) - No abstract available

Monotherapy • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Introduction to AMVUTTRA® (vutrisiran) (HFSA 2026) - "Sponsored by Alnylam"

Introduction to AMVUTTRA® (vutrisiran) (HFSA 2026) - "Sponsored by Alnylam"

IET 09: Industry Expert Theaters (No CEUs) - Introduction to AMVUTTRA® (vutrisiran) (HFSA 2026) - "For additional information about AMVUTTRA, please see the Important Safety Information and full Prescribing Information available at www.amvuttrahcp.com. Faculty Zubair Shah Gurusher Panjrath"

Amyloidosis • Cardiomyopathy • Cardiovascular • Musculoskeletal Pain • Pulmonary Disease

Effect of Vutrisiran According to Baseline Tafamidis Use in Transthyretin Amyloidosis with Cardiomyopathy (ESC 2026) - No abstract available

Late-breaking abstract • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Where evidence meets experience: redefining ATTR-CM treatment expectations (ESC 2026) - "Sponsored by Alnylam Pharmaceuticals To review the unmet need in ATTR-CM and how it may drive an evolution of the established treatment goals; To demonstrate the breadth of clinical data and outcomes from HELIOS-B and understand how vutrisiran can address the unmet need in ATTR-CM; and To share experts clinical experience and patient outcomes demonstrating how vutrisiran addresses the ambitious treatment goals in ATTR-CM...It will contain information on Alnylam medicinal products. May 2026 - AMV-INTX-00198"

Cardiovascular

Clinical evidence for vutrisiran in transthyretin cardiomyopathy (ESC 2026) - "Sponsored by Alnylam Pharmaceuticals"

Clinical • Cardiomyopathy • Cardiovascular

Cardiac presentation and treatment response by sex in patients with transthyretin amyloidosis: pooled analysis of vutrisiran and patisiran phase 3 studies (ESC 2026) - "In this pooled analysis of ATTR clinical studies, some baseline findings suggest more advanced disease in females vs males, despite relatively preserved myocardial function. Regardless of baseline differences, the pooled treatment effect of vutrisiran/patisiran versus placebo was generally consistent across sexes, supporting their potential for broad therapeutic applicability. Additional efforts are needed to improve earlier recognition of ATTR in women."

P3 data • Retrospective data • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Which therapy performs best across clinical domains in transthyretin amyloid cardiomyopathy? a frequentist network meta-analysis (ESC 2026) - "Patisiran showed favourable but less consistent signals across domains. Seven trials, including 2,494 patients, were analysed. No therapy consistently led all endpoints, supporting domain-specific heterogeneity. Vutrisiran showed the strongest biomarker and functional profile, with greater reductions in troponin I (SMD -3.65, 95% CI -3.90 to -3.40) and N-terminal pro-B-type natriuretic peptide (SMD -0.55, 95% CI -0.71 to -0.39), together with the largest improvement in 6-minute walk distance (SMD 0.53, 95% CI 0.24 to 0.82)."

Retrospective data • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Long-term efficacy of vutrisiran in patients with variant transthyretin amyloid cardiomyopathy (ESC 2026) - "Of these, 10 patients who initiated vutrisiran therapy were assigned to the vutrisiran-naïve group, whereas 8 patients who switched from patisiran to vutrisiran were assigned to the vutrisiran-switch group. Vutrisiran demonstrated favorable real-world clinical efficacy in patients with ATTRv-CM in our cohort."

Clinical • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Fewer adverse events reported in ATTR-CM patients treated with vutrisiran versus placebo: Post hoc safety analysis from HELIOS-B (ESC 2026) - " Exposure-adjusted event rates (per 100 patient-years [PY]) of AEs reported during the double-blind period (up to 36 months) of HELIOS-B were compared between vutrisiran and placebo arms in the overall and monotherapy populations (patients not receiving tafamidis at baseline), and baseline tafamidis subgroup. This post hoc analysis demonstrated that while patients with ATTR-CM experienced multiple AEs across SOCs, vutrisiran-treated patients had fewer AEs overall and across multiple systems. Among SOCs with the most frequent AEs, GI and nervous system disorders had the greatest difference with 42% and 41% fewer AEs in patients treated with vutrisiran, suggesting vutrisiran benefits extending beyond cardiac manifestations of ATTR."

Adverse events • Clinical • Retrospective data • Cardiomyopathy • Cardiovascular

Vutrisiran is associated with preservation of intrinsic capacity and healthy ageing in patients with transthyretin amyloidosis with cardiomyopathy: post hoc analysis of HELIOS-B (ESC 2026) - "The proportion of patients experiencing new impairment in these domains during the double-blind treatment period (up to 36 months) was assessed in patients receiving vutrisiran and placebo in the overall and monotherapy populations, and the baseline tafamidis subgroup. Vutrisiran was associated with a significant reduction in worsening of composite IC score and the majority of its domains vs placebo, with benefit to locomotion and nutritional status being relevant in ATTR-CM. Despite conclusions being limited by the use of unvalidated proxies for IC domains, these findings suggest that vutrisiran may help preserve functional reserve and support healthy ageing trajectories and IC in patients with ATTR-CM."

Clinical • Retrospective data • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Singapore is the first ASEAN country to provide government subsidy for eligible patients receiving vutrisiran for transthyretin amyloidosis (The Manila Times) - "HELIOS-A and B provide strong clinical evidence supporting improved patient outcomes...The treatment cost will be subsidised by up to 75% for eligible patients undergoing treatment with vutrisiran as a monotherapy."

Commercial • Amyloidosis • Cardiomyopathy

Combination Therapy in ATTR Cardiomyopathy: Hypothesis-Generating, Not Yet Practice-Changing. (PubMed, J Am Coll Cardiol) - No abstract available

Journal • Amyloidosis • Cardiomyopathy • Cardiovascular

Effect of Vutrisiran According to Baseline Tafamidis Use in Transthyretin Amyloidosis With Cardiomyopathy: Insights From HELIOS-B. (PubMed, J Am Coll Cardiol) - P3 | "Treatment effect estimates for vutrisiran on clinical outcomes were consistent across baseline tafamidis strata, with no statistically significant interaction according to baseline tafamidis use, with reduced effect size noted in those receiving baseline stabilizers in comparison to monotherapy. These data underscore the need for future prospective studies examining combination therapy in this population. (HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy; NCT04153149)."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Influence of Disease-Modifying Therapy on the Efficacy of Vutrisiran in Transthyretin Cardiac Amyloidosis. (PubMed, J Am Coll Cardiol) - P3 | "In HELIOS-B, there was no statistically significant evidence that the treatment benefit of vutrisiran on all-cause mortality and recurrent cardiovascular events was modified by concomitant use of tafamidis or heart failure therapies. These findings support the consistency of vutrisiran's efficacy across the spectrum of contemporary ATTR-CM pharmacotherapy. (HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy; NCT04153149)."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Targeted Therapy for ATTR-CM Amidst a Rapidly Evolving Landscape: A Moving Goalpost? (PubMed, J Am Coll Cardiol) - No abstract available

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Congestive Heart Failure • Heart Failure

Right Ventricular Function, Clinical Outcomes, and Effect of Vutrisiran in Transthyretin Amyloidosis With Cardiomyopathy: Secondary Analysis of the HELIOS-B Randomized Clinical Trial. (PubMed, JAMA Cardiol) - P3 | "This study found that, consistent with its beneficial effects on other measures of cardiac structure and function, vutrisiran stabilized RVFWS and improved RVFWS/PASP at 30 months. ClinicalTrials.gov Identifier: NCT04153149."

Clinical • Clinical data • Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Left Atrial Structure and Function, Clinical Outcomes, and Efficacy of Vutrisiran in Transthyretin Amyloidosis With Cardiomyopathy: A Secondary Analysis of the HELIOS-B Randomized Clinical Trial. (PubMed, JAMA Cardiol) - P3 | "Consistent with its beneficial effects on other measures of cardiac structure and function, vutrisiran attenuated worsening in LA strain at 30 months, supporting the importance of LA function in the pathophysiology of ATTR-CM and the ability of silencer therapy with vutrisiran to attenuate worsening atrial myopathy in amyloid heart disease. ClinicalTrials.gov Identifier: NCT04153149."

Clinical • Clinical data • Journal • Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Myositis