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Last Updated: February 16, 2025

CLINICAL TRIALS PROFILE FOR TAFAMIDIS


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All Clinical Trials for Tafamidis

Trial ID Title Status Sponsor Phase Start Date Summary
NCT00925002 ↗ Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis Completed Pfizer Phase 3 2009-08-05 This is a Phase 3, open-label study designed to obtain additional long-term safety and efficacy data for oral tafamidis (20 mg soft gelatin capsule) administered once daily (QD). In addition, this study continued to provide tafamidis to Val30Met subjects who had completed Protocol Fx-006 (a 1-year, open-label extension study to Protocol Fx-005 which was a randomized, double-blind, placebo-controlled, 18-month study to evaluate the safety and efficacy of tafamidis) or non-Val30Met subjects who had completed Protocol Fx1A-201 (a Phase 2, open-label study to evaluate TTR stabilization, safety, and tolerability of tafamidis) for up to 10 years or until subjects had access to tafamidis for ATTR-PN via prescription. Upon regulatory approval for the treatment of ATTR-PN in their respective country and access to prescription tafamidis, subjects may have been withdrawn from the study. Such subjects were considered study completers.
NCT00935012 ↗ Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy Completed Pfizer Phase 3 2009-09-30 Open-label Safety and Efficacy Evaluation of Fx-1006a in Patients with V122i Or Wild-type Transthyretin (ttr) Amyloid Cardiomyopathy. Patients who successfully complete Fx1B-201 will report to the clinical unit on Day 0 to sign the informed consent form and determine eligibility for Protocol Fx1B-303. In addition, on Day 0, patients will have their entrance criteria reviewed, and medical histories and demographic characteristics obtained. The physical examination (including weight and vital signs) and the relevant end of study clinical laboratory tests (alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, blood urea nitrogen, gamma glutamyl transferase, creatinine, total bilirubin, international normalized ratio, troponin I, troponin T, and amino-terminal B-type natriuretic peptide) from Protocol Fx1B-201 will be used for Protocol Fx1B-303. If more than 30 days has elapsed between the final study visit of Protocol Fx1B-201 and Day 0 of Protocol Fx1B-303, an abbreviated physical examination (including weight and vital signs) and clinical laboratory assessments must be performed on Day 0. Eligible patients will begin once-daily dosing with 20 mg Fx-1006A at home on Day 1 (i.e., first dose) and will return to the clinical unit for study visits every 6 months. Adverse events (AEs) and concomitant medication use will be collected at each 6-month visit to the clinical unit. Blood draws for clinical safety laboratory tests and abbreviated physical examinations (including weight and vital signs) will also be performed at each 6-month clinic visit. ECGs will be performed every 12 months on an annual basis. A telephone call will be made at 3-month intervals between clinic visits to assess safety and use of concomitant medications. For the evaluation of efficacy, the Patient Global Assessment, NYHA classification, KCCQ, 6-minute walk test, and efficacy-related clinical laboratory tests (serum levels of troponin T, troponin I, and NT-pro-BNP) will be determined every 6 months. In addition, echocardiograms will be performed every 12 months on an annual basis. An end of study visit including all safety and efficacy assessments will occur upon patient completion of the study, premature withdrawal (for any reason), or in the event of program discontinuation by the Sponsor.
NCT01369836 ↗ Study Of Single Doses Of PF-06291826 (Tafamidis) In Japanese And Western Subjects Completed Pfizer Phase 1 2011-07-01 The purpose of this study is the following: - To evaluate the safety, tolerability, and pharmacokinetics (PK) of orally administered tafamidis in Japanese and Western healthy volunteers at single dose. - To compare Japanese and Western PK profiles. - Determine the PD stabilization effect of tafamidis on human transthyretin (TTR) in a validated ex vivo assay.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for Tafamidis

Condition Name

Condition Name for Tafamidis
Intervention Trials
Healthy 9
Healthy Volunteers 5
Transthyretin (TTR) Amyloid Cardiomyopathy 2
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Condition MeSH

Condition MeSH for Tafamidis
Intervention Trials
Amyloidosis 11
Cardiomyopathies 9
Polyneuropathies 3
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Clinical Trial Locations for Tafamidis

Trials by Country

Trials by Country for Tafamidis
Location Trials
United States 75
Belgium 16
Canada 12
Japan 8
China 8
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Trials by US State

Trials by US State for Tafamidis
Location Trials
Massachusetts 5
Maryland 5
New York 5
Illinois 4
Connecticut 4
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Clinical Trial Progress for Tafamidis

Clinical Trial Phase

Clinical Trial Phase for Tafamidis
Clinical Trial Phase Trials
Phase 4 5
Phase 3 6
Phase 1 17
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Clinical Trial Status

Clinical Trial Status for Tafamidis
Clinical Trial Phase Trials
Completed 19
Not yet recruiting 6
Recruiting 3
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Clinical Trial Sponsors for Tafamidis

Sponsor Name

Sponsor Name for Tafamidis
Sponsor Trials
Pfizer 23
Corino Therapeutics, Inc. 1
Boston University 1
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Sponsor Type

Sponsor Type for Tafamidis
Sponsor Trials
Industry 25
Other 5
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Tafamidis: A Comprehensive Update on Clinical Trials, Market Analysis, and Projections

Introduction to Tafamidis

Tafamidis, marketed by Pfizer as Vyndaqel and Vyndamax, is a groundbreaking medication designed to treat transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and severe cardiovascular disease. Here, we delve into the clinical trials, market analysis, and future projections for this critical drug.

Clinical Trials: ATTR-ACT and Beyond

ATTR-ACT Trial

The ATTR-ACT trial was a pivotal phase 3, randomized, double-blind, placebo-controlled clinical trial that evaluated the efficacy and safety of tafamidis in patients with transthyretin amyloid cardiomyopathy. The trial enrolled 441 patients, with 264 receiving tafamidis and 177 receiving a placebo, over a 30-month period.

  • Primary Outcomes: The trial showed that tafamidis significantly reduced all-cause death, with 29.5% mortality in the tafamidis group compared to 42.9% in the placebo group (p < 0.05)[1][4].
  • Secondary Outcomes: Tafamidis also reduced cardiovascular-related hospitalizations and slowed the decline in functional capacity, as measured by the 6-minute walk test and the Kansas City Cardiomyopathy Questionnaire–Overall Summary test (p < 0.001 for each)[1][4].

Long-Term Efficacy and Safety

Long-term data from Pfizer's studies indicate that tafamidis delays disease progression in patients with hereditary transthyretin amyloid polyneuropathy (TTR-FAP). An analysis of up to 6 years of data showed that patients who started tafamidis early in the disease had less disease progression compared to those who started on placebo[3].

Market Analysis and Projections

Current Market Dominance

Pfizer's tafamidis has been the dominant treatment for ATTR-CM, with a strong market presence. Analysts from Leerink Partners predict that tafamidis will maintain a 60%-plus share of treated patients in the next few years, despite upcoming competition. The market for ATTR-CM is expected to grow significantly, from $2.4 billion in 2022 to nearly $4 billion by 2029[2].

Competition from BridgeBio

BridgeBio's acoramidis, another TTR stabilizer, is poised to enter the market. While acoramidis has shown promising results, including an 81% survival rate compared to 74% in the placebo group and a significant reduction in cardiovascular-related hospitalizations, it faces an uphill battle against the well-established tafamidis[5].

  • Physician Survey: A survey of physicians indicated that while some believe acoramidis is incrementally better, the majority see similar efficacy between acoramidis and tafamidis. Only a small percentage believe acoramidis is significantly better[5].

Market Growth and Projections

The ATTR-CM market is valued at $5.2 billion in 2023 and is projected to grow to $9.4 billion by 2031. Tafamidis is expected to continue its strong market performance, although the entry of acoramidis could introduce some competition. However, Pfizer's established brand and extensive clinical data are likely to maintain its market lead[2][5].

Clinical Benefits and Patient Outcomes

Improved Efficacy Measures

Tafamidis has been shown to improve various efficacy measures, including:

  • 6-Minute Walk Test: Patients treated with tafamidis had a better overall 6-minute walk test distance compared to those on placebo[4].
  • Kansas City Cardiomyopathy Questionnaire: Tafamidis-treated patients showed significant improvements in the Overall Summary score, indicating better quality of life[4].
  • Cardiac Function: Tafamidis improved measures of cardiac function, such as left ventricular ejection fraction (LVEF), LV global longitudinal strain, and E/e’ ratios[1].

Patient-Reported Outcomes

The Kansas City Cardiomyopathy Questionnaire–Overall Summary test and patient global assessment of overall health score demonstrated that tafamidis-treated patients had better patient-reported outcomes compared to those on placebo. These improvements are crucial for the quality of life and overall health of patients with ATTR-CM[4].

Mechanism of Action

Tafamidis works by binding to transthyretin, preventing its tetramer dissociation and subsequent amyloidosis. This mechanism is critical in stabilizing the TTR protein and reducing the deposition of amyloid in the heart, thereby slowing the progression of the disease[1][3].

Future Outlook and Challenges

Competition and Market Dynamics

While tafamidis is expected to maintain its market dominance, the entry of acoramidis and potentially other competitors could alter market dynamics. The ability of tafamidis to continue its strong performance will depend on its clinical superiority, physician preference, and Pfizer's marketing strategies[2][5].

Regulatory and Clinical Landscape

The FDA approval process for new treatments, such as acoramidis, will play a significant role in shaping the market. Ongoing and future clinical trials will continue to provide valuable data on the efficacy and safety of these treatments, influencing both physician and patient preferences[5].

Key Takeaways

  • Clinical Efficacy: Tafamidis has demonstrated significant clinical benefits in reducing mortality, hospitalizations, and functional decline in patients with ATTR-CM.
  • Market Dominance: Despite upcoming competition, tafamidis is expected to maintain a strong market presence due to its established brand and extensive clinical data.
  • Future Competition: The entry of acoramidis and other potential treatments will introduce competition, but tafamidis's clinical superiority and market presence are likely to sustain its lead.
  • Patient Outcomes: Tafamidis improves patient-reported outcomes and quality of life, making it a valuable treatment option for ATTR-CM patients.

FAQs

What is the primary mechanism of action of tafamidis?

Tafamidis works by binding to transthyretin, preventing its tetramer dissociation and subsequent amyloidosis, thereby reducing the deposition of amyloid in the heart.

What were the key findings of the ATTR-ACT trial?

The ATTR-ACT trial showed that tafamidis significantly reduced all-cause death, cardiovascular-related hospitalizations, and functional decline in patients with transthyretin amyloid cardiomyopathy.

How does tafamidis compare to acoramidis in terms of efficacy?

While acoramidis has shown promising results, the majority of physicians surveyed believe that tafamidis and acoramidis have similar efficacy, with only a small percentage believing acoramidis is significantly better.

What is the projected market growth for ATTR-CM treatments?

The ATTR-CM market is expected to grow from $5.2 billion in 2023 to $9.4 billion by 2031.

What are the potential challenges for tafamidis in the future market?

The entry of new competitors, such as acoramidis, and ongoing clinical trials could introduce competition and alter market dynamics, although tafamidis's established brand and clinical superiority are expected to sustain its market lead.

Sources

  1. Tafamidis in Transthyretin Cardiomyopathy Clinical Trial - ATTR-ACT. American College of Cardiology.
  2. Pfizer's 'entrenched' tafamidis franchise will be tough to challenge. FiercePharma.
  3. Amyloid Publishes Long-Term Data Analysis from Pfizer Suggesting Tafamidis Delays Progression of TTR-FAP. Pfizer.
  4. Improvements in Efficacy Measures With Tafamidis in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial. JACC Journals.
  5. BridgeBio Wins FDA Approval for ATTR-CM Drug, Launching Tight Race With Pfizer. BioSpace.

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