New Real-World Data Published in Journal of Cardiac Failure on Effectiveness of VYNDAQEL (tafamidis) in Patients with Transthyretin Cardiac Amyloidosis
August 8, 2024 – Drug Discovery, Other, Pharmaceutical – Pfizer, cardiac failure
Patients treated with tafamidis were associated with greater rates of survival compared with patients untreated with tafamidis.
6 August 2024 — Walton Oaks, UK — Pfizer Ltd today announced publication of real-world results from a cohort of the longitudinal, observational Phase 4 THAOS (Transthyretin Amyloidosis Outcomes Survey) study evaluating survival rates among patients with transthyretin cardiac amyloidosis (ATTR-CM) treated or untreated with tafamidis in the Journal of Cardiac Failure.
The pivotal phase 3 ATTR-ACT (Transthyretin Amyloid Cardiomyopathy Clinical Trial) demonstrated that tafamidis improved survival and reduced the risk of cardiovascular-related hospitalization compared with placebo in patients with ATTR-CM. Real-world evidence complements randomized controlled trials by providing key insights into diverse clinical settings and patient populations.
In this real-world cohort from the THAOS study, survival rates were numerically higher than reported in the ATTR-ACT clinical trial, and no new safety signals were identified. These results from THAOS add further support for the effectiveness of tafamidis for patients with ATTR-CM.
“It’s encouraging to see improved survival rates among ATTR-CM patients in a real-world setting, suggesting that earlier diagnosis and treatment with tafamidis could be associated with better outcomes for patients” said Pablo Garcia-Pavia, M.D., Ph.D., Hospital Universitario Puerta de Hierro Majadahonda, CNIC and CIBERCV, Madrid, Spain, and lead author of the THAOS ATTR-CM survival analysis. “These real-world results from this important disease registry help us gain additional insight into the natural course of ATTR-CM, including the impact of treatment intervention, which is especially valuable for making informed decisions in the care of these patients.”
The THAOS ATTR-CM survival analysis included a total of 1,441 patients with ATTR-CM from 18 countries enrolled between 2007 and 2023. In the tafamidis-treated study arm, 77.5% received tafamidis meglumine 80 mg or tafamidis free acid 61 mg throughout the study. The remaining patients received tafamidis meglumine 20 mg (not licensed for the treatment of ATTR-CM) throughout the study (8.9%), initially received tafamidis meglumine 20 mg then switched to tafamidis meglumine 80 mg/tafamidis free acid 61 mg (12.4%) or received another dose (1.2%).The licensed dose for tafamidis in ATTR-CM is 61 mg once daily. Tafamidis 61 mg corresponds to 80 mg tafamidis meglumine. Results showed:
- Survival rates (95% CI) at 30 and 42 months, respectively, were 84.4% (80.5-87.7) and 76.8% (70.9-81.7) in tafamidis-treated patients (n=587), and 70.0% (66.4-73.2) and 59.3% (55.2-63.0) in tafamidis-untreated patients (n=854).
- Survival rates were higher in a contemporary cohort as reflected by a sensitivity analysis performed on patients enrolled after 2019 versus prior to 2019.
- No new safety signals were identified in the tafamidis-treated cohort. All-causality, treatment-emergent adverse events (TEAEs) occurred in 27.4% tafamidis-treated patients, which was numerically lower than the percentage of tafamidis-treated patients who experienced TEAEs in ATTR-ACT.
“ATTR cardiac amyloidosis is a life limiting condition and is an underecognised cause of heart failure,” said Seema Patel, Medical Director, Specialty Care, Pfizer UK. “These results from THAOS further support the findings from the ATTR-ACT trial on tafamidis which show it has the potential to address the unmet medical need that exists for people living with this rare type of cardiac amyloidosis.”
Real-world analyses assess how a medicine performs in routine medical practice and can only evaluate association and not causality. The analyses have the potential to supplement clinical trial data by providing additional information about how a medicine performs in routine medical practice. Real-world data analyses cannot be used as stand-alone evidence to validate the efficacy and/or safety of a treatment.
The completion of the THAOS registry marks an important milestone for the tafamidis program, adding to more than a decade of research. THAOS (NCT00628745) is the largest and longest completed ATTR-amyloidosis disease registry to date, enrolling a total of 6,718 participants over a 16-year period from 2007 to 2023.
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