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

Introduction to AMVUTTRA® (vutrisiran) (ACC 2026) - No abstract available

Precision medicine with pioneering RNAi therapeutics in ATTR amyloidosis: from bench to bedside. (PubMed, Eur J Intern Med) - "Major clinical trials evaluating first- and second-generation RNA-targeted agents-including patisiran, vutrisiran, inotersen, and eplontersen-are summarized, highlighting efficacy, safety profiles, dosing regimens, and relevance across different ATTR phenotypes. As RNA-based therapies continue to evolve, integration of molecular insights with clinical phenotyping and real-world evidence will be essential to fully realize the potential of precision medicine for patients with ATTR amyloidosis."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Developmental Disorders • Pain

Quality of life and effectiveness of vutrisiran as treatment for hereditary transthyretin amyloidosis. (PubMed, Farm Hosp) - "Although statistical significance was not reached, vutrisiran maintained or improved quality of life and clinical status in a real-world population that was more pretreated and had greater disease burden than the clinical trial. Combined with its favorable safety profile, subcutaneous administration, and extended dosing interval-preferred by patients-vutrisiran represents a promising therapeutic option for ATTRv amyloidosis. Long-term real-world studies are warranted to confirm these findings."

HEOR • Journal • Amyloidosis • Cardiac Amyloidosis • Rare Diseases

VUTRISIRAN REDUCES THE RISK OF DEVELOPING ADVANCED DISEASE AND DEMONSTRATES BENEFIT IN PATIENTS WHO DEVELOP ADVANCED DISEASE IN ATTR-CM: ANALYSIS FROM THE HELIOS-B STUDY (ACC 2026) - "Abstract is embargoed at this time."

Clinical • Metastases • Cardiovascular

IMPACT OF VUTRISIRAN ON THE COMPONENTS OF QUALITY OF LIFE IN TRANSTHYRETIN CARDIOMYOPATHY (ATTR-CM) (ACC 2026) - "Abstract is embargoed at this time."

HEOR • Cardiomyopathy • Cardiovascular

Cardiac Biomarkers as an Early Warning System in Transthyretin Cardiac Amyloidosis. (PubMed, J Am Coll Cardiol) - No abstract available

Biomarker • Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Efficacy of Suppression of Serum Transthyretin With Patisiran and Vutrisiran in Variant ATTR Amyloidosis: An Observational Crossover Study. (PubMed, Circulation) - No abstract available

Journal • Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Congestive Heart Failure • Heart Failure

Transthyretin Cardiac Amyloidosis Evaluation and Management: 2025 ACC Concise Clinical Guidance. (PubMed, J Am Coll Cardiol) - "Finally, individuals with transthyretin amyloid cardiomyopathy have benefitted from advances in broadly effective heart failure therapies, namely mineralocorticoid receptor antagonists and sodium glucose-cotransporter 2 inhibitors, as well as specific disease-modifying therapies with transthyretin stabilizers, tafamidis and acoramidis, and the transthyretin silencer vutrisiran. The purpose of this Concise Clinical Guidance is to offer updated strategies to clinicians, reflecting the expanding therapeutic landscape, and reinforcing best practices for the diagnosis and management of transthyretin amyloid cardiomyopathy with a focus on choice of disease-modifying therapies, heart failure therapies, and future directions."

Clinical guideline • Journal • Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Congestive Heart Failure • Heart Failure • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Pain • Transplantation

Current and Future Treatment Landscape of Transthyretin Amyloid Cardiomyopathy. (PubMed, Cardiol Ther) - "Therapeutic advances have significantly increased treatment possibilities, selection of appropriate therapy, switching between therapies, combination strategies, and how to monitor treatment response over time. This review summarizes available and investigational therapies for ATTR-CM and considers practical questions that guide clinical decision-making, with the goal of helping clinicians navigate the evolving therapeutic landscape."

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

4017. Introduction to AMVUTTRA® (vutrisiran) (ACC 2026) - "Industry-Expert Theater presentations are not part of ACC.26, as planned by its Program Committee, and do not qualify for continuing medical education (CME), continuing nursing education (CNE) or continuing education (CE) credit. Sponsored by Alnylam Pharmaceuticals"

Amyloidosis • Cardiomyopathy • Cardiovascular

ADHERENCE AND PERSISTENCE WITH VUTRISIRAN, AN RNAI THERAPEUTIC FOR THE TREATMENT OF TRANSTHYRETIN AMYLOIDOSIS (ACC 2026) - "Abstract is embargoed at this time."

Adherence • Amyloidosis • Cardiac Amyloidosis

EVALUATION OF VUTRISIRAN IN ATTR AMYLOIDOSIS WITH CARDIOMYOPATHY (ACC 2026) - "Abstract is embargoed at this time."

Amyloidosis • Cardiomyopathy • Cardiovascular

Impact of Disease-Modifying Therapies on Imaging Parameters in Cardiac Amyloidosis: A Systematic Review and Meta-Analysis. (PubMed, Curr Probl Cardiol) - "NMA in ATTR cardiac amyloidosis showed that patisiran (MD -1.04%, 95% CI -1.56 to -0.52), tafamidis (-0.91%, -1.40 to -0.41), and vutrisiran (-0.90%, -1.75 to -0.05) improved GLS versus placebo, compared to acoramidis. Disease-directed therapy in ATTR and hematologic response in AL cardiac amyloidosis are associated with preservation or improvement of GLS and LV wall thickness, supporting their use as surrogate endpoints for treatment monitoring."

Journal • Retrospective data • Review • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Hematological Disorders

POST HOC ANALYSIS OF DIASTOLIC DYSFUNCTION IN THE HELIOS-B STUDY OF VUTRISIRAN IN PATIENTS WITH TRANSTHYRETIN AMYLOIDOSIS WITH CARDIOMYOPATHY (ACC 2026) - "Abstract is embargoed at this time."

Clinical • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Cholesterol-dependent control of endosomal escape regulates intracellular trafficking of small interfering RNA therapeutics and interactions with small molecule drugs. (PubMed, J Pharmacol Exp Ther) - "Notably, chloroquine, an endosomal escape enhancer, rescues gene silencing under cholesterol-reduced conditions...SIGNIFICANCE STATEMENT: This study uncovers cholesterol as an essential and previously unrecognized determinant of small interfering RNA therapeutic efficacy, acting through annexin A2 to enable endosomal escape, a critical bottleneck in RNA drug delivery. The findings position cholesterol modulation as a viable approach to improve the intracellular delivery and therapeutic effectiveness of RNA-based drugs."

Journal • Metabolic Disorders • ANXA2

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (clinicaltrials.gov) - P3 | N=125 | Recruiting | Sponsor: Alnylam Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Amyloidosis • Cardiac Amyloidosis • Pain

MyocardON-TTR: Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran (clinicaltrials.gov) - P=N/A | N=20 | Active, not recruiting | Sponsor: Rennes University Hospital | Recruiting ➔ Active, not recruiting | Trial completion date: Jul 2026 ➔ Dec 2027 | Trial primary completion date: Feb 2026 ➔ Dec 2027

Enrollment closed • Trial completion date • Trial primary completion date • Amyloidosis • Cardiac Amyloidosis • Pain

Alnylam Announces Health Canada Approval of AMVUTTRA (vutrisiran), the First and Only RNAi Therapeutic for the Treatment of Cardiomyopathy in Adult Patients with ATTR Amyloidosis (Canada Newswire) - "The Health Canada approval was based on positive results from the pivotal HELIOS-B Phase 3 study – a randomized, double-blind, placebo-controlled, multicenter, global trial that enrolled adult patients with wtATTR or hATTR amyloidosis with cardiomyopathy."

Canada approval • Amyloidosis • Cardiomyopathy

REPLY: Vutrisiran in ATTR-CM: Questions on Combination Therapy and Early Biomarker Kinetics. (PubMed, J Am Coll Cardiol) - No abstract available

Biomarker • Journal

Small RNA or oligonucleotide drugs and challenges in evaluating drug-drug interactions. (PubMed, Front Pharmacol) - "Widespread adoption of these strategies has further enabled the application of oligonucleotides as viable drugs and expanded the class of RNA therapeutics, with thirteen antisense oligonucleotides (ASOs) (fomiversen, mipomersen, nusinersen, inotersen, eteplirsen, golodirsen, casimersen, viltolarsen, tofersen, eplontersen, olezarsen, and donidalorsen), seven small interfering RNAs (siRNAs) (patisiran, givosiran, lumasiran, inclisiran, vutrisiran, nedosiran, and fitusiran), and two aptamers (pegaptanib and avacincaptad pegol) that have been approved by the United States Food and Drug Administration (FDA). This article provides an overview of FDA-approved oligonucleotide therapies, emphasizing chemical modifications, molecular targets for mechanistic actions, and available ADME and PK/PD properties, followed by the discussion of critical needs for risk assessment strategies suited for this unique modality that focuses on possible DDIs with concomitant drugs. The latter may..."

Journal • Review

Evaluation of Vutrisiran in ATTR Amyloidosis with Cardiomyopathy (ASHP 2025) - No abstract available

Amyloidosis • Cardiomyopathy • Cardiovascular

From Stabilization to Silencing: Assessing Adherence and Outcomes of Tafamidis and Vutrisiran in Patients with Cardiac Amyloidosis in a Rural Setting (ASHP 2025) - No abstract available

Adherence • Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiovascular

Stabilizers, Silencers, and Disease Stage: Lessons From Recent Trials of ATTR Cardiac Amyloidosis. (PubMed, J Am Coll Cardiol) - No abstract available

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Vutrisiran in ATTR-CM: Questions on Combination Therapy and Early Biomarker Kinetics. (PubMed, J Am Coll Cardiol) - No abstract available

Biomarker • Journal

Class-Specific Adverse Events of Patients Treated with Small Interfering RNA Therapeutics: A Disproportionality Analysis of the United States Food and Drug Administration Adverse Event Reporting System Database Based on the MY FAERS Platform. (PubMed, Nucleic Acid Ther) - "This study aimed to identify and quantify CAE-siRNA associated with U.S. Food and Drug Administration (FDA)-approved siRNA drugs (patisiran, givosiran, vutrisiran, inclisiran, and lumasiran) using real-world pharmacovigilance data, focusing on potential class-wide effects. This study identified clinically relevant CAE-siRNA, particularly hepatic toxicity for inclisiran, supporting enhanced monitoring. While disproportionality analyses are hypothesis generating, these findings underscore the need for targeted pharmacovigilance to optimize the safety of this promising drug class."

Adverse events • Journal • Back Pain • Fatigue • Gastroenterology • Gastrointestinal Disorder • Musculoskeletal Pain • Pain