Tafamidis 61mg capsules

Tafamidis, a transthyretin (TTR) kinetic stabilizer, delayed neuropathic progression in patients with Val30Met TTR familial amyloid polyneuropathy (TTR-FAP) in an 18-month randomized controlled trial (study Fx-005). This 12-month, open-label extension study evaluated the long-term safety, tolerability, and efficacy of tafamidis 20 mg once daily in 86 patients who earlier received blinded treatment with tafamidis or placebo. Efficacy measures included the Neuropathy Impairment Score in the Lower Limbs (NIS-LL), Norfolk Quality of Life-Diabetic Neuropathy total quality of life (TQOL) score, and changes in neurologic function and nutritional status. We quantified the monthly rates of change in efficacy measures, and TTR stabilization, and monitored adverse events (AEs). Patients who continued on tafamidis had stable rates of change in NIS-LL (from 0.08 to 0.11/month; p = 0.60) and TQOL (from -0.03 to 0.25; p = 0.16). In patients switched from placebo, the monthly rate of change in NIS-LL declined (from 0.34 to 0.16/month; p = 0.01), as did TQOL score (from 0.61 to -0.16; p < 0.001). Patients treated with tafamidis for 30 months had 55.9 % greater preservation of neurologic function as measured by the NIS-LL than patients in whom tafamidis was initiated later. Plasma TTR was stabilized in 94.1 % of patients treated with tafamidis for 30 months. AEs were similar between groups; no patients discontinued because of an AE. Long-term tafamidis was well tolerated, with the reduced rate of neurologic deterioration sustained over 30 months. Tafamidis also slowed neurologic impairment in patients previously given placebo, but treatment benefits were greater when tafamidis was begun earlier

Abstract licence: CC BY 2.0

BackgroundWe used network meta-analyses to evaluate the pharmacological interventions for Hereditary Transthyretin-related Amyloidosis with Polyneuropathy (ATTRv-PN).MethodsWe searched Medline, Embase, and Cochrane (June 2025) for randomized trials assessing pharmacological interventions in ATTRv-PN adults. Two reviewers independently screened, extracted data, and assessed risk of bias. Primary efficacy outcomes were mNIS+7 and Norfolk-QoL-DN. Primary safety outcome was serious adverse events (SAE). We used Bayesian hierarchical models. Evidence certainty was assessed using GRADE.ResultsSix trials (n = 989) were included (3 at high-risk of bias). Participant ages and disease duration ranged from 52.8 to 62.0 and 1.4 to 3.9 years, respectively. For mNIS+7, data were available for all interventions except tafamidis. All demonstrated statistically significant improvements versus placebo. Vutrisiran (standardized mean difference [SMD] vs. placebo: -1.66; 95% credible interval [CrI]: -2.13 to -1.17) and patisiran (SMD vs. placebo: -1.56; 95% CrI: -1.88 to -1.25) demonstrated improvements compared with diflunisal, eplontersen, and inotersen. For Norfolk-QoL-DN, data were available for all interventions except diflunisal. All except tafamidis demonstrated statistically significant improvements versus placebo. Patisiran (MD vs. placebo: -17.39; 95% CrI: -23.22 to -11.57), vutrisiran (MD vs. placebo: -16.99; 95% CrI: -25.24 to -8.72), and eplontersen (MD vs. placebo: -15.56; 95% CrI: -21.97 to -9.15) demonstrated improvements compared with tafamidis. For SAE, there were no differences between active interventions versus placebo. Confidence in the evidence varied from very low to moderate.ConclusionGene-silencing therapies were more efficacious, although these findings should be regarded as hypothesis-generating given the scarcity of data, lack of head-to-head trials, and clinical heterogeneity across trials.

Abstract licence: CC BY-NC-ND

Background: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive infiltrative cardiomyopathy associated with substantial morbidity and mortality. This systematic review evaluates the effects of tafamidis on echocardiographic parameters, cardiac biomarkers, functional outcomes, mortality, and safety in patients with ATTR-CM. Methods: This systematic review was conducted in accordance with PRISMA 2020 guidelines and registered in PROSPERO (CRD420261391553). PubMed, Embase, and Web of Science were searched through 20 March 2026. Eligible studies included adults with ATTR-CM treated with tafamidis and reporting, echocardiographic, biomarker, functional, mortality, or safety outcomes. Randomized controlled trials and observational studies were included. Risk of bias was assessed using the Cochrane RoB 2 tool and Newcastle-Ottawa Scale. Due to substantial heterogeneity, a narrative synthesis was performed. Results: Seventeen studies were included with a total of 1890 patients. Tafamidis treatment was potentially associated with stabilization of global longitudinal strain, preservation of functional status, and lower all-cause mortality compared with untreated or control cohorts. Biomarker findings, including N-terminal pro-B-type natriuretic peptide (NT-proBNP) and high-sensitivity cardiac troponin T (hs-cTnT), were heterogeneous and did not demonstrate a consistent pattern of improvement. Functional outcomes, including New York Heart Association (NYHA) class, 6-minute walk distance (6MWT), National Amyloidosis Center (NAC) staging, and quality-of-life measures, suggested slower clinical deterioration among treated patients. Limited available safety data indicated that tafamidis was generally well tolerated, with no major safety concerns identified. Conclusions: Current evidence may suggest that tafamidis slows disease progression and may potentially be associated with improved survival in ATTR-CM. Further prospective studies with standardized outcome reporting are needed. Evidence was limited by heterogeneity in study design, outcome reporting, and follow-up duration, with most included studies being observational.

Abstract licence: CC BY