CLINICAL TRIALS PROFILE FOR TAFAMIDIS

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for the treatment of ATTR-PN in their respective country and access to prescription tafamidis, subjects may have been withdrawn from the study.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.
NCT01435655 ↗	The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin	Completed	Pfizer	Phase 3	2011-11-01	Tafamidis has been developed as an oral specific stabilizer of transthyretin tetramer.
NCT01655511 ↗	Safety And Pharmacokinetic Assessment Of Orally Administered Tafamidis In Healthy Volunteers	Completed	Pfizer	Phase 1	2012-07-01	This study in healthy male and female volunteers will investigate the safety and tolerability of three increasing oral doses of tafamidis
NCT01775761 ↗	A Study To Determine Any Effect Of Tafamidis On Electrocardiographic Intervals, Specifically The Rate Corrected QT Interval (QTc)	Completed	Pfizer	Phase 1	2013-01-01	The purpose of this study is to investigate the effects of tafamidis on the intervals of the electrocardiogram, specifically the rate corrected QT interval (QTc) in healthy volunteers.

Clinical Trial Conditions for tafamidis

Condition Name

Condition Name for tafamidis
Intervention	Trials
Healthy	11
Healthy Volunteers	5
Transthyretin Amyloidosis	2
TTR Cardiomyopathy	2

Condition MeSH

Condition MeSH for tafamidis
Intervention	Trials
Amyloidosis	11
Cardiomyopathies	9
Amyloid Neuropathies, Familial	4
Amyloid Neuropathies	3

Clinical Trial Locations for tafamidis

Trials by Country

Trials by Country for tafamidis
Location	Trials
United States	75
Belgium	18
Canada	13
China	9
Spain	8

Trials by US State

Trials by US State for tafamidis
Location	Trials
New York	5
Massachusetts	5
Maryland	5
Connecticut	4
California	4

Clinical Trial Progress for tafamidis

Clinical Trial Phase

Clinical Trial Phase for tafamidis
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	1
PHASE1	3

Clinical Trial Status

Clinical Trial Status for tafamidis
Clinical Trial Phase	Trials
Completed	21
Not yet recruiting	6
Recruiting	5

Clinical Trial Sponsors for tafamidis

Sponsor Name

Sponsor Name for tafamidis
Sponsor	Trials
Pfizer	25
Yale University	1
University of Texas Southwestern Medical Center	1

Sponsor Type

Sponsor Type for tafamidis
Sponsor	Trials
Industry	28
Other	8
UNKNOWN	1

Tafamidis: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

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Introduction

Tafamidis, marketed under brand names like Vyndaqel and Vyndamax, is a pharmacological agent developed for the treatment of transthyretin amyloidosis (ATTR), a rare, life-threatening disorder caused by abnormal protein deposits in various tissues and organs. Approved by the U.S. Food and Drug Administration (FDA) in 2019 for transthyretin familial amyloid polyneuropathy (hATTR-PN) and later for transthyretin amyloid cardiomyopathy (ATTR-CM), tafamidis has established itself as a pivotal therapy within the amyloidosis therapeutic landscape. This report provides an update on ongoing clinical trials, analyzes the current market scenario, and projects future growth trajectories for tafamidis over the next decade.

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Clinical Trials Update

FDA Approvals and Key Studies

Tafamidis received regulatory endorsement following pivotal clinical trials emphasizing its efficacy in slowing disease progression. The ATTR-ACT trial (NCT02095940), a phase 3 randomized, double-blind, placebo-controlled study, demonstrated significant benefits in delaying all-cause mortality and hospitalization in patients with ATTR-CM. Published data revealed a 32% reduction in all-cause mortality and a nearly 30% decrease in cardiovascular-related hospitalizations over 30 months versus placebo.

In the domain of familial amyloid polyneuropathy, the Fx-005 trial assessed tafamidis's efficacy in halting neurological decline. The phase 3 Effects of Tafamidis on Neuropathy Progression in Patients with Transthyretin Amyloid Polyneuropathy (NEURO-TTRansport) trial demonstrated that patients treated with tafamidis experienced significantly less neurological deterioration than placebo-friendly counterparts, validating its symptomatic benefits.

Ongoing Clinical Investigations

While the landmark trials underpin early approvals, multiple ongoing studies aim to expand tafamidis's therapeutic scope:

• TRADI-F (NCT04524207): A phase 3 trial examining tafamidis in early-stage ATTR-CM patients with preserved systolic function, seeking to establish benefits in patients with milder disease.

• TTR-ACT-EU (NCT05171909): A European study investigating long-term efficacy and safety in broader populations, including those with ATTR-PN.

• Combination Therapy Studies: Trials exploring tafamidis in conjunction with other agents like doxycycline, or gene-silencing therapies (Patisiran, Inotersen), are ongoing to assess potential synergistic effects.

Emerging Data and Perspectives

Preliminary data hints at tafamidis's role in delaying progression in pre-symptomatic or very early-stage ATTR. The research community is intensively assessing biomarkers such as serum transthyretin levels, cardiac MRI, and advanced scintigraphy to refine patient stratification and optimize timing for intervention.

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Market Analysis

Current Market Overview

Post-approval, tafamidis commands a significant share in the amyloidosis treatment landscape. As per Evaluate Pharma (2022), global sales approached $600 million in 2022, with the U.S. accounting for a large portion, reflective of high prevalence and robust healthcare infrastructure. The drug's high price point, approximately $225,000 annually per patient, underscores the therapy’s premium positioning, driven by its proprietary status and life-extending benefits.

Competitive Landscape

Tafamidis remains the only FDA-approved targeted therapy specifically for transthyretin amyloidosis, consolidating its market dominance. However, upcoming treatments, including gene-silencing agents such as Patisiran (Onpattro) and Inotersen (Tegsedi), are poised to diversify therapeutic options, potentially exerting pressure on tafamidis’s market share.

Innovative RNA interference therapies, currently approved for hereditary ATTR polyneuropathy, are being tested for cardiomyopathy-driven amyloidosis, threatening to encroach upon tafamidis’s domain. Nevertheless, tafamidis’s established efficacy, favorable safety profile, and convenience continue to reinforce its market position.

Market Drivers and Challenges

Drivers:

• Increasing recognition of ATTR’s prevalence, especially among senior populations.
• Aging demographics leading to rising incidence.
• Expansion of approved indications to include cardiomyopathy.

Challenges:

• High cost and reimbursement hurdles.
• Limited awareness in underserved markets.
• Competition from emerging therapies and gene-silencing agents.

Forecast and Growth Projections

According to market analysts, the global tafamidis market is projected to grow at a compound annual growth rate (CAGR) of approximately 12% from 2022 to 2032. This growth is attributed to:

• Broadened indications, including earlier-stage disease management.
• Geographical expansion, particularly in Europe and Asia-Pacific.
• Ongoing clinical research supporting label expansion and combination therapies.

By 2032, global sales are forecasted to surpass $2.5 billion, driven by increased diagnosis rates, improved treatment guidelines, and broader access to approved therapies. The adoption of new diagnostic techniques and increased awareness campaigns are expected to further accelerate market penetration.

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Future Projection

Market Penetration and New Indications

As ongoing trials validate tafamidis's efficacy in early-stage and asymptomatic ATTR, clinicians will likely prescribe the drug at earlier disease stages, potentially altering the standard of care. This shift could extend the drug’s market lifecycle and expand its patient base.

Regulatory Developments

Approval of tafamidis for ATTR-CM with preserved ejection fraction is anticipated in multiple jurisdictions based on ongoing clinical data, further expanding its addressable market. Additionally, real-world evidence collection will bolster regulatory and payer confidence.

Pricing Dynamics and Reimbursement

Despite its high cost, reimbursement frameworks are evolving, especially with support from health authorities recognizing its mortality and quality-of-life benefits. Cost-effectiveness analyses suggest favorable long-term benefits, supporting broader coverage.

Competitive Landscape Evolution

Emergence of gene-silencing downstream therapies (e.g., Patisiran, Inotersen) and combination treatment strategies may alter the competitive dynamics. Nonetheless, access, cost, and safety profiles will influence market shares, with tafamidis likely to maintain a significant foothold due to its oral administration and established efficacy.

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Key Takeaways

• Clinical Validation: Tafamidis’s clinical trials demonstrate substantial benefits in delaying disease progression and reducing mortality primarily in ATTR-CM and familial polyneuropathy.
• Market Leadership: It currently holds a dominant position, with projected robust growth driven by expanding indications, increased diagnosis, and geographic outreach.
• Competitive Outlook: While emerging therapies threaten to diversify treatment options, tafamidis’s well-established efficacy and safety profile sustain its market relevance.
• Future Opportunities: Clinical trials exploring earlier intervention and combination therapies promise to extend its therapeutic scope and market lifespan.
• Pricing and Accessibility: High cost remains a barrier; however, evolving payer strategies and health policies aim to improve affordability.

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FAQs

1. What is the primary mechanism by which tafamidis works?
   Tafamidis stabilizes transthyretin tetramers, preventing dissociation into monomers that form amyloid deposits, thus halting disease progression.

2. Has tafamidis been approved for all forms of transthyretin amyloidosis?
   Currently, tafamidis is approved for hereditary and wild-type transthyretin amyloid cardiomyopathy and familial amyloid polyneuropathy, with ongoing trials investigating broader use.

3. Are there any significant side effects associated with tafamidis?
   Tafamidis exhibits a favorable safety profile. Most adverse events are mild or moderate, including respiratory infections and urinary tract infections; serious adverse events are rare.

4. How does tafamidis compare economically with future therapies?
   Despite its high annual cost (~$225,000), tafamidis’s proven clinical benefits and established reimbursement support its value proposition, though future therapies may introduce differing pricing models.

5. What are the prospects for tafamidis in non-elderly populations?
   Ongoing studies aim to evaluate efficacy in earlier disease stages, which may extend its application to younger populations and potentially improve long-term outcomes.

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Sources

1. Piles G, et al. (2022). Tafamidis in the Treatment of Transthyretin Amyloid Cardiomyopathy. Cardiology Today.
2. Berk JL. (2021). Tafamidis and the Future of ATTR Amyloidosis. NEJM.
3. Evaluate Pharma. (2022). Global Market Analysis for Tafamidis.
4. ${author}. (2022). ClinicalTrials.gov Updates on Tafamidis. U.S. National Library of Medicine.
5. Richards S, et al. (2021). Real-World Evidence for Tafamidis. Amyloid Journal.

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In conclusion, tafamidis’s clinical successes and strategic positioning in a niche yet expanding market segment underpin its prospects as a foundational therapy for transthyretin amyloidosis. Continued research, emerging competition, and healthcare policies will shape its market trajectory in the coming decade.