Vyndaqel (tafamidis meglumine) / Pfizer - LARVOL DELTA

Shifting symptom profiles and accelerated diagnosis in AL amyloidosis: Findings from ARC community surveys 2022–2024 (ASH 2025) - "Tafamidis, the first FDA-approved amyloidosis therapy fortransthyretin cardiac amyloidosis in 2019, sparked an increased awareness of amyloid-relatedcardiomyopathies, including AL amyloidosis. In 2021, with daratumumab as the first FDA-approvedtherapy specific to AL amyloidosis, further disease-specific AL amyloidosis awareness and educationincreased... Patients with AL amyloidosis recently diagnosed from 2022-2024 experienced shorterdiagnostic delays but continue to see multiple physicians prior to diagnosis and initiating treatment. Thismay suggest once patients are in the health care system physicians can quickly diagnose, but the multi-step referral process may continue to contribute to delays. The shift in symptom profiles suggests arising disease awareness and better screening allowing patients to receive treatment faster."

Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Impact of High-Dose Tafamidis on Hereditary ATTR (ATTRv) Amyloidosis With Central Nervous System Involvement: Two Case Reports With Clinical, Radiological and Cerebrospinal Fluid Follow Up. (PubMed, J Peripher Nerv Syst) - "In our two patients, progression of CNS disease occurred whilst on high-dose tafamidis, highlighting the importance of further studies into the monitoring and treatment of CNS manifestations of ATTRv."

Journal • Amyloidosis • CNS Disorders • Epilepsy • Pain

Tailoring Anesthesia for Cardiac Amyloidosis: A Case Report Highlighting Perioperative Challenges. (PubMed, Cureus) - "Anticoagulation was reversed with idarucizumab, and perioperative hemodynamic instability was managed with vasopressors and dynamic fluid responsiveness assessment. Furthermore, the authors highlight the perioperative hematologic, metabolic, and electrolyte disorders that may arise from disease and cardiac-target therapies such as tafamidis and sodium-glucose cotransporter-2 inhibitors (SGLT2 inhibitors). We emphasize the importance of individualized planning, multidisciplinary coordination, and vigilant perioperative monitoring to optimize outcomes in this high-risk population."

Journal • Amyloidosis • Anesthesia • Atrial Fibrillation • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Chronic Kidney Disease • CNS Disorders • Depression • Heart Failure • Hematological Disorders • Movement Disorders • Musculoskeletal Diseases • Nephrology • Orthopedics • Parkinson's Disease • Psychiatry • Renal Disease

Discontinuation of Tafamidis in Wild-Type Transthyretin Amyloid Cardiomyopathy Patients. (PubMed, Circ Rep) - "The period from tafamidis discontinuation to death was shorter in ATTRwt-CM patients hospitalized for non-cardiovascular diseases. Discussions of the right period for discontinuation are needed."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • 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

Pyrazole-Based Transthyretin Kinetic Stabilizers Identified Using a Covalent Fluorescent Probe Assay for Selectivity Profiling in Human Serum. (PubMed, J Med Chem) - "Among these, 3,5-dichloropyrazole derivatives exhibited efficacy comparable to that of tafamidis and acoramidis. X-ray crystallography of the TTR-17 complex confirmed hydrogen bonding with Ser117/117' and electrostatic interactions with Lys15. Pharmacokinetic studies of compounds 16 and 17 demonstrated favorable exposure, bioavailability, and metabolic stability, supporting their preclinical development for hereditary- and wild-type TTR amyloidosis."

Journal • Amyloidosis

Real-World Efficacy of Tafamidis in Korean ATTR-CM Patients: A Retrospective Observational Strain Analysis. (PubMed, Korean Circ J) - "Tafamidis was associated with improved survival and slower disease progression in Korean ATTR-CM. LASI and RV-pulmonary circulation coupling index may serve as sensitive markers of treatment response."

Journal • Real-world evidence • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Concurrent light chain and transthyretin cardiac amyloidosis: A case report and review of the literature. (PubMed, Medicine (Baltimore)) - "We report a confirmed case of coexistent AL-CA and wild-type transthyretin cardiac amyloidosis, validated through comprehensive diagnostic evaluation. Although dual therapy targeting both amyloid types may be needed, the lack of guidelines and follow-up data highlights the need for standardized management approaches. Future studies should prioritize long-term outcome tracking."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Congestive Heart Failure • Fatigue • Heart Failure • Hematological Disorders • Pulmonary Disease

Methods of identifying transthyretin amyloid cardiomyopathy in secondary data sources: a systematic review. (PubMed, Curr Med Res Opin) - "Sixteen analyses (61.5%) used International Classification of Diseases-10th-Revision (ICD-10) codes, 23.1% used ICD-9 and -10 codes, 11.5% analyses used country unique billing codes, and 11.5% relied on tafamidis prescriptions (with/without billing codes) to identify ATTR-CM...Methods for identifying ATTR-CM in studies of secondary datasets were heterogeneous. Future research should focus on optimizing ATTR-CM identification algorithms and performing validation studies."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Hematological Malignancies • Oncology

Misfolded Fate: A Case Report of Transthyretin Cardiac Amyloidosis in Ghana. (PubMed, Cureus) - "The patient was treated with a sodium-glucose cotransporter-2 inhibitor (SGLT2i) and a mineralocorticoid receptor antagonist (MRA), without disease-modifying therapy such as tafamidis. This case highlights cardiac amyloidosis in a female patient and the feasibility of the non-biopsy pathway for diagnosing transthyretin cardiac amyloidosis in Ghana and across the broader African region. This condition is significantly underrecognized in the sub-region, despite the expectation that genetic variants are common."

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

Cost-effectiveness of vutrisiran, tafamidis, and their combination in amyloid transthyretin cardiomyopathy. (PubMed, Eur Heart J Qual Care Clin Outcomes) - No abstract available

HEOR • Journal • Cardiomyopathy • Cardiovascular

SGLT2 inhibitors and Tafamidis in ATTR-CM: Promise or premature optimism? (PubMed, J Cardiol) - No abstract available

Journal

Role SGLT2 inhibitor therapy in patients with transthyretin cardiac amyloidosis: a GRADE assessed systematic review and meta-analysis. (PubMed, Acta Cardiol) - "Transthyretin amyloid cardiomyopathy (ATTR-CM) patients continue to experience worsening heart failure despite therapy with disease modifying therapies (tafamidis, patisiran, etc.). However, these findings are derived from observational studies with their inherent biases and must be interpreted with caution. High-quality randomised controlled trials are needed to confirm these associations and better define their clinical role in ATTR-CM."

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

Global Longitudinal Strain for Prognostic Staging in Wild-Type Transthyretin Cardiac Amyloidosis. (PubMed, Circ Cardiovasc Imaging) - "Likelihood ratio test indicated incremental prognostic value of GLS (staging) over baseline NAC staging (P<0.001). GLS is a strong, independent mortality predictor in ATTRwt-CM, irrespective of tafamidis treatment, that may be an adjunct or complementary to biomarker staging."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Tafamidis Reduces Death and Hospitalization for Acute Heart Failure in Octogenarian Patients With Transthyretin Cardiac Amyloidosis: A Propensity Score-Weighted Cohort Study. (PubMed, J Am Heart Assoc) - "Tafamidis led to a lower mortality rate and fewer hospital readmissions for acute heart failure in octogenarian patients with transthyretin cardiac amyloidosis."

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

Changing Patterns of Diagnosis and Survival in Transthyretin Cardiac Amyloidosis: A Multicenter Cohort Study. (PubMed, J Am Heart Assoc) - "In this multicenter study of patients seen at referral centers for transthyretin cardiac amyloidosis, diagnosis in a more recent era was associated with improved survival, while tafamidis benefits were attenuated by disease severity and older age. Significant residual mortality risk remains in treated patients at higher disease stages."

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

Relative efficacy of tafamidis, acoramidis, patisiran and vutrisiran in patients with transthyretin cardiac amyloidosis: a network meta-analysis. (PubMed, Eur Heart J Open) - "When the ATTR-ACT trial was excluded from the analysis, vutrisiran monotherapy consistently showed the highest probability of being ranked better than other treatments in terms of primary end-point. Although differences in trial design and study populations complicate direct efficacy comparisons, tafamidis demonstrated the highest efficacy in improving survival, reducing cardiovascular hospitalizations, and enhancing functional capacity and quality of life in patients with ATTR-CA, but also vutrisiran and acoramidis emerged as viable options."

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

Real-world effectiveness of targeted therapies in ATTR cardiomyopathy: A meta-analysis integrating population-based data. (PubMed, ESC Heart Fail) - "Targeted treatments for ATTR-CM significantly reduce mortality and cardiovascular hospitalizations. When extrapolated to population-level data, these treatments show clinical benefits, emphasizing the importance of early diagnosis and therapeutic intervention."

Journal • Real-world evidence • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Clinical phenotype and prognosis of real-world patients with wild-type transthyretin amyloid cardiomyopathy treated with tafamidis. (PubMed, Eur J Heart Fail) - "In this large, contemporary, real-world cohort of patients with ATTRwt-CM, predominantly in NYHA class I or II, treatment with tafamidis was consistently associated with a significantly lower risk of all-cause mortality."

Journal • Real-world evidence • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

A real-world study of tafamidis in people with transthyretin amyloid cardiomyopathy (ATTR-CM) with heart and nerve symptoms: a plain language summary. (PubMed, Future Cardiol) - No abstract available

Journal • Real-world evidence • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

A Study to Learn About the Study Medicine Called Tafamidis 61mg in People Diagnosed With Transthyretin Amyloid Cardiomyopathy (ATTR-CM) (clinicaltrials.gov) - P=N/A | N=13 | Completed | Sponsor: Pfizer | Recruiting ➔ Completed | Trial completion date: Apr 2026 ➔ Oct 2025 | Trial primary completion date: Apr 2026 ➔ Oct 2025

Real-world evidence • Trial completion • Trial completion date • Trial primary completion date • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

A Study to Learn About Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in India (clinicaltrials.gov) - P=N/A | N=6 | Completed | Sponsor: Pfizer | Recruiting ➔ Completed | N=15 ➔ 6

Enrollment change • Trial completion • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Timing of Mortality Benefit in Outcomes Trials in Transthyretin Amyloidosis. (PubMed, J Am Coll Cardiol) - "TTR silencers and stabilizers in ATTR-CM confer a delayed but consistent mortality benefit, with no significant differences observed between the three major trials. This uniform pattern may reflect a shared mechanism of action-reducing new amyloid deposition rather than reversing established disease, and underscore the importance of early treatment initiation and adequate trial duration to capture delayed mortality effects."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Vutrisiran Improved Outcomes Versus Placebo in Patients with Transthyretin Amyloidosis with Cardiomyopathy and Severe Chronic Kidney Disease: Post Hoc Analysis of HELIOS-B (AHA 2025) - P3 | "Outcomes were assessed overall and by baseline tafamidis use (monotherapy and baseline tafamidis subgroups). Median (IQR) eGFR at baseline in pts receiving vutrisiran and PBO was 64 (50–81) and 65 (53–81) mL/min/1.73m2, respectively. Vutrisiran appeared to preserve renal function in pts with ATTR-CM. Consistent with results from the overall population, vutrisiran reduced the risk of ACM and CV events vs PBO in pts with ATTR-CM and advanced CKD in HELIOS-B; results require corroboration in a larger pt population."

Clinical • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Chronic Kidney Disease • Nephrology • Renal Disease

Patterns of Treatment Adherence and Administration Preferences for People With Transthyretin Amyloid Cardiomyopathy: A Real-World Survey in Europe, the United States, and Japan (ISPOR-EU 2025) - "At survey, 73% of patients were prescribed treatment, of which 93% were prescribed DMTs including tafamidis (85%), patisiran (6%), vutrisiran (2%) and diflunisal (2%). Tafamidis was the most frequently prescribed DMT, and adherence was high. Patients more frequently reported feeling comfortable with oral medications than injections/infusions, particularly once daily orals, underscoring the relevance of administration preferences among treatment landscapes. Further research is needed to explore outcomes with ATTR treatments."

Adherence • Clinical • Real-world • Real-world evidence • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular