Vyndaqel (tafamidis meglumine) / Pfizer - LARVOL DELTA

Cardiac amyloidosis: From diagnosis to therapeutics breakthroughs. (PubMed, World J Exp Med) - "AL amyloidosis is primarily managed with chemotherapy and, when appropriate, stem cell transplantation, while tafamidis is currently the only approved treatment for ATTR amyloidosis, though new therapeutic agents are in development. Overall, recent progress in diagnosis and treatment has enhanced patient outcomes, and early detection combined with coordinated care is key to managing this complex condition."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Transplantation

Associations of Tafamidis With New Atrial Fibrillation Risk in Transthyretin Cardiomyopathy. (PubMed, JACC Clin Electrophysiol) - No abstract available

Journal • Atrial Fibrillation • Cardiomyopathy • Cardiovascular

Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy (clinicaltrials.gov) - P=N/A | N=50 | Completed | Sponsor: Pfizer | Active, not recruiting ➔ Completed

Real-world evidence • Trial completion • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders

NEUROPROTECTIVE POTENTIAL OF A NOVEL, MODIFIED TAFAMIDIS PRODRUG IN A MOUSE MODEL OF SPORADIC ALZHEIMER'S DISEASE (ADPD 2026) - "These findings demonstrate region-selective neuroprotective properties of TA, particularly in the hippocampus. However, regional variability and contradictory brainstem effects underscore the complex, incompletely understood role of TA in the central nervous system, necessitating further investigation to elucidate therapeutic potential and mechanisms in Alzheimer's disease. This work was financed under the Pfizer Inc."

Preclinical • Alzheimer's Disease • Amyloidosis • CNS Disorders • Inflammation • GFAP • IGF1R

Genomics in Child Health and the APS/SPR Mary Ellen Avery Neonatal Research Award (PAS 2026) - "It has also led to therapy outside of gene replacement, such as the CFTR modulars for cystic fibrosis (Trikafta), hydroxyurea for sickle cell disease or hereditary amyloidosis (tafamidis). Discuss the controversies around genome sequencing for universal newborn screening. Understand genetic mechanisms for increased susceptibility to Herpes Simplex infections."

Clinical • Amyloidosis • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Hematological Disorders • Herpes Simplex • Immunology • Infectious Disease • Respiratory Diseases • Sickle Cell Disease

SAFETY AND EFFICACY OF ACORAMIDIS IN TRANSTHYRETIN AMYLOID CARDIOMYOPATHY: A SYSTEMATIC REVIEW AND META-ANALYSIS (ACC 2026) - "Acoramidis demonstrates substantial TTR stabilization and a favorable safety profile, though modest functional, clinical and biomarker improvements were observed. Despite high heterogeneity, these findings support acoramidis as a promising therapeutic option for ATTR-CM. Further head-to-head studies with tafamidis are warranted."

Retrospective data • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

RARE COEXISTENCE OF LIGHT-CHAIN AND TRANSTHYRETIN CARDIAC AMYLOIDOSIS: A CASE OF DUAL PATHOLOGY (ACC 2026) - "He was treated with CyBorD+daratumumab and tafamidis, and remained stable at 6 months Decision-Making: This case highlights the rare coexistence of AL and ATTRv CA, a dual-hit process of light-chain toxicity and TTR infiltration. Concurrent AL and ATTRv CA is rare but clinically significant. Vigilance for discordant findings, confirmatory biopsy, and tailored therapy are key to improving outcomes"

Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

A CASE OF WILD-TYPE TRANSTHYRETIN CARDIAC AMYLOIDOSIS MIMICKING HYPERTROPHIC CARDIOMYOPATHY (ACC 2026) - "The patient was started on Tafamidis... CA should be considered in patients with unexplained LV wall thickening. Early and accurate diagnosis is essential, as timely initiation of disease-modifying therapy may improve outcomes."

Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

TAFAMIDIS AND GDMT IN TRANSTHYRETIN CARDIAC AMYLOIDOSIS: SURVIVAL BENEFITS AND HOSPITALIZATION REDUCTION IN PRACTICE (ACC 2026) - "In ATTR-CA, addition of Tafamidis to GDMT yields substantial and durable reductions in mortality and hospitalizations, confirming its role as cornerstone therapy. The addition of GDMT to Tafamidis may confer complementary long-term survival benefit, supporting a Tafamidis-first approach with additive value of GDMT warranting prospective validation."

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

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) - P3 | "Endpoints included composite ACM and recurrent cardiovascular events through 33-36 months (primary endpoint) and ACM through 42 months (secondary endpoint) in the overall and monotherapy (patients not on tafamidis at baseline) populations, and adverse events. Fewer patients receiving vutrisiran in HELIOS-B developed advanced disease vs placebo. Among these, the risk of composite ACM and recurrent cardiovascular events and ACM were lower and there were fewer adverse events with vutrisiran vs placebo."

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

ADHERENCE AND PERSISTENCE WITH VUTRISIRAN, AN RNAI THERAPEUTIC FOR THE TREATMENT OF TRANSTHYRETIN AMYLOIDOSIS (ACC 2026) - "Until recently, tafamidis was the only approved treatment for ATTR cardiomyopathy (ATTR-CM). Real-world adherence to vutrisiran was excellent, with high persistence over 12 months. These findings may inform clinical decision-making in ATTR-CM. Further research directly comparing adherence and persistence among ATTR-CM therapies is needed"

Adherence • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy

WHEN RARE MEETS RARE: COEXISTENCE OF QUADRICUSPID AORTIC VALVE AND CARDIAC AMYLOIDOSIS (ACC 2026) - "He was started on Tafamidis... Severe AS in young patients is typically due to bicuspid AV and rarely QAV. Multimodality imaging (CT, MRI) helps identify the cause. AS diagnosis is challenging when LVH leads to LVOT obstruction, as it affects trans-AV gradients."

Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Coronary Artery Disease • Heart Failure • Pulmonary Disease

REVERSAL OF STRAIN ABNORMALITIES IN WILD-TYPE TRANSTHYRETIN CARDIAC AMYLOIDOSIS: A CASE REPORT (ACC 2026) - "This case highlights tafamidis-associated recovery of strain abnormalities in wild-type ATTR-CM, underscoring the importance of early recognition, timely therapy, and advanced imaging for monitoring response, while expanding expectations beyond stabilization to potential recover"

Case report • Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Congestive Heart Failure • Coronary Artery Disease • Dyslipidemia • Heart Failure • Hypertension

TREATMENT PATTERNS AND PREFERENCES IN TRANSTHYRETIN AMYLOID CARDIOMYOPATHY IN THE UNITED STATES: RESULTS FROM A REAL-WORLD SURVEY (ACC 2026) - "Tx was received by 60.5% of pts, 6.6% had prior tx, and 32.8% were tx naive; 86.5% received TTR stabilizers (83.2% tafamidis, 3.2% diflunisal, 0% acoramidis). Physicians were not fully satisfied with tx for >55% of pts prescribed. Pts were most comfortable with oral therapy."

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

PROGNOSTIC IMPLICATIONS OF ECHOCARDIOGRAPHIC PARAMETERS IN PATIENTS WITH TRANSTHYRETIN CARDIAC AMYLOIDOSIS ON TAFAMIDIS (ACC 2026) - "Despite the mortality benefit of tafamidis in ATTR-CA, elevated PCWP and bilateral pressures may be associated with worse outcomes. Further studies are needed to evaluate the utility of cardiac imaging to identify high-risk phenotypes."

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

AUTOINFLAMMATORY PERICARDITIS IN TAFAMIDIS-TREATED WILD-TYPE TRANSTHYRETIN AMYLOIDOSIS: MRI-DOCUMENTED REMISSION WITH IL-1 INHIBITION (ACC 2026) - "Decision-Making: Rilonacept induced rapid symptom relief, CRP normalization to <3 mg/L, and successful taper of steroids and colchicine without relapse. This case illustrates that ATTRwt-CM can be complicated by inflammatory pericarditis, identifiable by serial MRI. Phenotype-directed therapy, including IL-1 inhibition, may achieve durable remission even in advanced amyloidosis. To our knowledge, this is the first reported case of MRI-documented, IL-1-responsive pericarditis in a tafamidis-treated ATTRwt patient."

Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Inflammation

FUNCTIONAL STABILITY MEASURED WITH 6MWD IN PATIENTS TREATED WITH TAFAMIDIS VS PLACEBO: POST-HOC ANALYSIS FROM THE TAFAMIDIS IN TRANSTHYRETIN CARDIOMYOPATHY CLINICAL TRIAL (ACC 2026) - P3 | "Tafamidis resulted in a higher proportion of patients with functional stability after 1 year of treatment, with a significantly lower risk of ACM during the subsequent 18 months of follow-up. In patients with 6MWD progression, tafamidis significantly reduced the risk of CVH. This underscores the importance of treatment in maintaining functional stability and improving outcomes for patients with worsening heart failure."

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

RNA INTERFERENCE THERAPEUTICS REDUCE CARDIAC ARRHYTHMIAS AND ARRHYTHMIC HOSPITALISATIONS IN NON-TAFAMIDIS USERS WITH TRANSTHYRETIN AMYLOIDOSIS: A META-ANALYSIS OF RANDOMISED CONTROLLED TRIALS (ACC 2026) - "In this meta-analysis of RCTs, RNAi therapeutics significantly reduced cardiac arrhythmias and arrhythmic hospitalisations in patients who were non-users of tafamidis."

Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiovascular

MORTALITY IN OCTAGENARIAN PATIENTS WITH TRANSTHYRETIN AMYLOIDOSIS TREATED WITH TAFAMIDIS. A SYSTEMATIC REVIEW AND METANALYSIS. (ACC 2026) - "These findings suggest that tafamidis use in octagenarians patients with transthyretin amyloidosis is associated with significant reduction in all-cause mortality. Further randomized controlled trials are warranted to confirm these results."

Clinical • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy

HOCM OR ATTR?: PERSISTENT DIAGNOSTIC AMBIGUITY AFTER ADVANCED IMAGING (ACC 2026) - "Despite a technically successful myectomy, his symptoms persisted, and Tafamidis therapy was initiated after much discussion... This case illustrates the unique challenge of biopsy confirmed myocardial ATTR in the absence of symptoms and cardiac imaging findings. The mismatch between pathology and phenotype raises uncertainty about whether amyloid was incidental or contributory to HOCM pathology."

Metastases • Amyloidosis • Cardiac Amyloidosis • CNS Disorders • Obstructive Hypertrophic Cardiomyopathy • Pulmonary Disease

BEYOND LIGHT CHAINS: THE OVERLOOKED BURDEN OF TTR AMYLOIDOSIS IN PLASMA CELL DYSCRASIAS (ACC 2026) - "The patient remained clinically stable on tafamidis, while continuing teclistamab therapy for MM. TTR amyloidosis should be considered even with high suspicion for AL amyloidosis. Evidence shows TTR is more prevalent than previously recognized and can coexist with light-chain disorders. Recognizing this overlap is critical to avoid misclassification, allowing timely disease-modifying therapy, and improved outcomes."

Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Malignancies • Monoclonal Gammopathy • Multiple Myeloma • Pulmonary Disease

INCIDENCE AND OUTCOMES OF DE NOVO ATRIAL FIBRILLATION IN TRANSTHYRETIN AMYLOID CARDIOMYOPATHY PATIENTS RECEIVING TAFAMIDIS (ACC 2026) - "Tafamidis was not associated with reduced de novo atrial fibrillation in ATTR-CM, suggesting its benefits do not extend to arrhythmia prevention. However, treatment significantly lowered all-cause mortality, indicating a survival advantage likely driven by disease stabilization rather than rhythm control. These findings support tafamidis as a disease-modifying therapy that improves survival, highlighting the need for continued arrhythmia monitoring alongside treatment."

Clinical • Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

PROSPECTIVE EVALUATION OF LOCALIZED WILD-TYPE ATTR DEPOSITION IN MUSCULOSKELETAL TISSUES AND ITS RELATIONSHIP TO CARDIAC AMYLOIDOSIS (ACC 2026) - "Tafamidis therapy was initiated in this case... Localized TTR deposition in musculoskeletal tissues may, in some patients, progress to cardiac amyloidosis. The concept of purely localized ATTRwt as an independent condition appears limited. These findings underscore the need for long-term cardiovascular surveillance and suggest that orthopedic tissue analysis may provide an opportunity for early detection of cardiac amyloidosis."

Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics

LONG-TERM SURVIVAL AFTER LIVER TRANSPLANT IN VAL30MET HEREDITARY TRANSTHYRETIN AMYLOIDOSIS (ACC 2026) - "While tafamidis offers disease modification, liver transplantation (LTx) remains an option in select genotypes, with >50% of Val30Met carriers surviving up to 20 years... LTx remains a valid disease-modifying option in Val30Met ATTRv patients with rapid deterioration despite pharmacologic therapy. Early genetic diagnosis and timely transplant referral optimize survival and functional stability."

Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Monoclonal Gammopathy • Pulmonary Disease • Transplantation

IMPACT OF TAFAMIDIS ON STROKE RISK IN ATTR CARDIAC AMYLOIDOSIS WITH ATRIAL FIBRILLATION ON DOAC THERAPY (ACC 2026) - "In patients with ATTR-CA and AF on DOAC therapy, tafamidis use was associated with significant reduction in ischemic stroke risk. These findings suggest tafamidis may protect this population beyond transthyretin stabilization alone."

Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiovascular • Ischemic stroke