Delving into the Latest Updates on Tafamidis with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

他伐米迪斯, PF-06291826-00, Vyndamax

+ [4]

Target

TTR

Action

modulators

Mechanism

TTR modulators(Transthyretin modulators)

Therapeutic Areas

Nervous System DiseasesCardiovascular DiseasesCongenital Disorders+ [2]

Active Indication

Transthyretin Amyloid CardiomyopathyAmyloidosis, Hereditary, Transthyretin-Related

Inactive Indication

Poikiloderma With NeutropeniaSenile cardiac amyloidosis

Originator Organization

Pfizer Inc.

Active Organization

FoldRx Pharmaceuticals LLC

Pfizer Inc.Pfizer Canada ULC

+ [4]

Inactive Organization-

License Organization-

Drug Highest PhaseApproved

First Approval Date

Japan (26 Mar 2019),

Amyloidosis, Hereditary, Transthyretin-Related

RegulationOrphan Drug (United States), Orphan Drug (European Union), Orphan Drug (South Korea), Orphan Drug (Australia)

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Structure/Sequence

Molecular FormulaC14H7Cl2NO3

InChIKeyTXEIIPDJKFWEEC-UHFFFAOYSA-N

CAS Registry594839-88-0

The purpose of this study is to learn about the safety of Tafamidis for the treatment of Transthyretin amyloid cardiomyopathy (ATTR-CM) in India.
ATTR-CM is a condition that affects people's hearts. Transthyretin is a protein that is made in the liver. In some people this protein stops working and forms clumps called as amyloid. Transthyretin amyloid builds up in heart and stops the heart from pumping properly.
This study is seeking for participants who are:
* confirmed with ATTR-CM.
* given Tafamidis capsules to be taken by mouth.
The safety of Tafamidis capsules will be checked based on side effects. These side effects can happen within 6 months after taking Tafamidis. A side effect is something (expected or unexpected) that you feel was caused by a medicine or treatment you take. The study doctor will collect side effect information and put the information on patient's case form.
Follow-up of the patient's will be performed via clinic re-visit or over a call. It is not a rule for the participants to visit the clinic in this study.
This study will help to see if Tafamidis is safe.

Start Date25 Mar 2024

Sponsor / Collaborator

Pfizer Inc.

100 Clinical Results associated with Tafamidis

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100 Translational Medicine associated with Tafamidis

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100 Patents (Medical) associated with Tafamidis

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578

Literatures (Medical) associated with Tafamidis

01 Dec 2025·Transplantation Reviews

Impact of disease-modifying drugs in patients with transthyretin amyloidosis after liver transplantation: a systematic review

Review

Author: Santo, Christiane ; Dabarian, Andre ; Romero, Cristhian ; Fernandes, Fabio ; Vaz Kerges Bueno, Bruno

BACKGROUND:

Orthotopic liver transplant (OLT) was the first approved treatment for hereditary transthyretin amyloidosis (ATTRv). However, some patients continue to deteriorate due to ongoing wild-type TTR deposition and residual synthesis from extrahepatic sources. In recent years, disease-modifying therapies including TTR stabilizers (e.g., Tafamidis) and gene-silencing agents (e.g., Patisiran) have emerged, but their role in post-OLT patients remains unclear due to their exclusion from most clinical trials.

METHODS:

A systematic search was conducted in PubMed, Cochrane, and Embase (up to June 2025) using terms related to transthyretin amyloidosis, liver transplantation, and disease-modifying therapies. The objective was to evaluate clinical benefits and safety of these agents in symptomatic ATTRv patients after OLT.

RESULTS:

Disease-modifying therapies showed potential benefits in post-OLT ATTR patients. A total of 39 patients treated with tafamidis, inotersen, or patisiran were analyzed. Neurological improvements, including autonomic symptoms, NIS score, and quality of life, were based on 3 case reports and 32 patients from observational studies. Cardiovascular results were from 4 case reports, and biomarker findings from 3 case reports.

CONCLUSIONS:

Disease-modifying therapies may offer clinical benefits in post-OLT ATTRv patients. However, robust prospective studies and randomized trials are needed to confirm efficacy and ensure safety in this population.

01 Dec 2025·MEDICINA CLINICA

Transthyretin stabilizer targeting for transthyretin amyloid cardiomyopathy: A systematic review and meta-analysis

Review

Author: Tavares de Melo, Marcelo Dantas ; Andrade Fernandes, João Vitor ; de Abrantes Formiga, Yan Gadelha ; de Lima Beltrão, Fabyan Esberard ; de Oliveira Ramos, João Victor ; de Oliveira Ferreira, Carlos Henrique

INTRODUCTION:

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive disease caused by cardiac amyloid deposition. Transthyretin stabilizers (TTRS) are a potential treatment, but their efficacy and safety remain uncertain. This study aims to evaluate the effects of TTRS on all-cause mortality, functional capacity, quality of life, and adverse events in ATTR-CM patients.

METHODS:

A systematic review and meta-analysis was conducted in February 2025 using Cochrane Central, PubMed, and Embase to identify clinical trials and observational studies comparing TTRS versus no-TTRS therapies. Two reviewers independently assessed study eligibility and extracted data. Outcomes were analyzed using odds ratios (OR) and mean differences (MD) with 95% confidence intervals. Heterogeneity was evaluated using I² statistics, and sensitivity analyses were performed where appropriate. Risk of bias was assessed using ROBINS-I V2 and RoB 2 tools.

RESULTS:

Seventeen studies with 5209 participants (2399 TTRS; 2810 controls) were included. TTRS significantly reduced all-cause mortality (OR 0.20; 95% CI 0.11-0.37; p<0.01). In the tafamidis subgroup, the effect remained significant (OR 0.25; 95% CI 0.13-0.48; p<0.01). No mortality benefit was observed in groups with mixed tafamidis doses (OR 0.75; 95% CI 0.41-1.38; p=0.14). TTRS improved quality of life (KCCQ-OS: MD 11.70) and functional capacity (6-minute walk test: SMD 50.68). Fewer adverse events (OR 0.19) and serious events (OR 0.54) were reported, with no difference in severe events.

CONCLUSION:

TTRS, especially tafamidis, reduce mortality and improve outcomes in ATTR-CM.

01 Dec 2025·Current heart failure reports

Etiological Treatment of Cardiac Amyloidosis: Standard of Care and Future Directions

Review

Author: Morfino, Paolo ; Emdin, Michele ; Vergaro, Giuseppe ; Chubuchna, Olena ; Aimo, Alberto ; Castiglione, Vincenzo ; Buda, Gabriele ; Ferrari Chen, Yu Fu ; Fabiani, Iacopo

Abstract:

Purpose of Review:

Cardiac amyloidosis (CA) is a condition caused by interstitial infiltration of misfolded proteins structured into amyloid fibrils. Transthyretin (ATTR) and immunoglobulin light chain (AL) amyloidosis represent the most common forms of CA. CA was traditionally perceived as a rare and incurable disease, but diagnostic and therapeutic advances have undermined the conventional paradigm.

Recent Findings:

The standard of care for ATTR-CA include agents capable of selectively stabilizing the precursor protein (e.g., tafamidis), whereas the plasma cell clone is the main target of chemotherapy for AL-CA. For long, tafamidis represented the only drug approved for patients with ATTR-CA. Recent data from ATTRibute-CM led to the approval of acoramidis, whereas patisiran received refusal based on the APOLLO-B trial. Novel CRISPR-Cas9-based drugs (i.e., NTLA-2001) hold great potential in the setting of ATTR-CA. Several hematological regimens are available to treat AL-CA. The main limit of current therapies is their inability to trigger removal of amyloid from tissues. However, the investigation of monoclonal antibodies targeting misfolded ATTR (e.g., PRX004, NI301A) or AL (e.g., birtamimab, anselamimab) has led to encouraging results.

Summary:

Various cutting-edge strategies are being tested for treatment of CA and may change the prognostic landscape of this condition in the next years.

166

News (Medical) associated with Tafamidis

01 Dec 2025

·www.fiercebiotech.com

Protego plans pivotal trial for AL amyloidosis prospect with $130M series B

Protego Biopharma\'s oversubscribed series B was led by Novartis Venture Fund and Forbion. \n Protego Biopharma is gearing up to bring its amyloid light-chain (AL) amyloidosis prospect into pivotal testing using its new $130 million series B fundraise.The oversubscribed funding round, led by Novartis Venture Fund and Forbion, picked up new support from Omega Funds, Droia Ventures, YK Bioventures and Digitalis Ventures. Existing investors including Vida Ventures, MPM BioImpact, Lightspeed Venture Partners and Scripps Research also pitched in, underscoring “the promise of our science and the urgency of our mission,” Protego’s CEO Brent Warner said in a Monday press release. With the fresh capital, the biotech is positioned to advance lead candidate PROT-001 into pivotal trials, bringing the biotech one step closer to delivering “the first disease-modifying therapy for AL amyloidosis and offering new hope to patients who currently face devastating outcomes,” according to Warner.AL amyloidosis is a rare and potentially fatal condition triggered by abnormal plasma cells that produce excess light chain proteins that misfold, forming fibrils that deposit in various organs and tissues—including the heart. Protego’s strategy to address the condition is rooted in human genetics and “unique pharmacological chaperone mechanism,” it said in the release.By stabilizing immunoglobulin light chains and therefore preventing amyloid buildup, PROT-001 is designed to directly tackle the disease as opposed to managing symptoms, representing what Protego calls a “potential paradigm shift not only for AL amyloidosis, but also for a wide spectrum of protein misfolding disorders with profound unmet needs.”“At Forbion, we invest in bold teams turning breakthrough science into tangible medical and commercial impact, and Protego exemplifies that vision,” Forbion principal Tim Lohoff, Ph.D., commented.San Diego-based Protego was established in 2017 by Jeffery Kelly, Ph.D., Richard Labaudinière, Ph.D., and Xin Jiang, Ph.D. The company attributes its approach in protein misfolding diseases to the work done by Kelly and Labaudinière discovering and developing tafamidis, a transthyretin amyloid cardiomyopathy drug that Pfizer now brands as Vyndaqel after its 2010 acquisition of Kelly and Labaudinière’s FoldRx.Besides AL amyloidosis, Protego is focused on protein misfolding that causes myopathy, cardiomyopathy, stroke, renal disease, retinal diseases, channelopathies and various degenerative diseases, it says. In 2021, investors contributed $51 million in a series A financing round to support the company’s mission. Protego thinks PROT-001 has the promise to succeed in an area where Big Pharmas and biotechs alike have previously stumbled. AstraZeneca’s anti-fibril antibody anselamimab, for one, missed its primary endpoint of all-cause mortality or frequency of cardiovascular hospitalizations in a phase 3 trial over the summer. The results proved a big blow to the program the company considered (PDF) a potential rare disease blockbuster after splashing out $150 million for it in its 2021 Caelum Biosciences buy. AZ’s flop came after similar failures for Prothena, which revived its AL amyloidosis candidate birtamimab after a 2018 miss that prompted mass layoffs. But, after a phase 3 study that read out in May and found Prothena’s antibody was no better in reducing time to all-cause mortality than placebo, the biotech finally gave up on the program and implemented more layoffs.

Phase 3Acquisition

01 Dec 2025

·endpoints.news

Protego gets $130M for AL amyloidosis drug from leaders behind Vyndaqel

A San Diego biotech hoping to crack into a rare protein misfolding disease has drummed up $130 million in investor support to get into a potential pivotal trial next year. Protego Biopharma, from the creators of Pfizer’s amyloidosis medicine Vyndaqel, has secured a Series B from lead investors Novartis Venture Fund and Forbion. Omega Funds, Droia Ventures, Vida Ventures, MPM BioImpact and others also chipped into the round, the company said on Monday morning. The biotech, founded in 2017 , is developing small molecules to reprogram protein folding. Its lead drug is for amyloid light chain amyloidosis, or AL amyloidosis. The rare disease is marked by a buildup of misfolded proteins in the heart and other organs. The toxic proteins can damage organs and lead to heart failure, sometimes resulting in death. Protego’s funding arrives just a few weeks after the FDA stamped full approval onto Johnson & Johnson’s Darzalex Faspro for the disease. The treatment received accelerated approval in January 2021 . Meanwhile, two high-profile clinical failures occurred earlier this year at Prothena and for AstraZeneca’s anselamimab , dealing a blow to the UK pharma’s $3 billion projections for the antibody. Protego’s candidate, nicknamed PROT-001, works to help proteins fold correctly, the company said. Essentially, Protego doesn’t want to address symptoms, but rather the main cause of the disease. “The standard of care is shutting down the plasma cells or trying to eradicate most of them. Having a light chain stabilizer at its source that can capture the availability of this misfolded light chain should lead to significant improvements in morbidity and mortality,” Protego CEO Brent Warner said in an interview with Endpoints News . PROT-001 is in a Phase 1 healthy volunteer trial in Australia, Warner said. The biotech anticipates collecting the traditional single-ascending and multiple-ascending dose data in the first half of 2026 and then filing for a Phase 2/3 trial later in the year, the CEO said. “Even with standard of care, patients who’ve been on it, even post-treatment, still have some of those plasma cells available that is continuously pumping out toxic light chain, and even a small amount of that toxic misfolded light chain is detrimental to cells,” Warner said. “Having a stabilizer that would be available to neutralize that toxicity should lead to no further formation of aggregates and allow those patients to get back into more of a native, natural state.” The biotech was co-founded by Scripps Research professor Jeffery Kelly, Xin Jiang and Richard Labaudinière. Kelly’s research previously led to the creation of FoldRx Pharmaceuticals, which was led by Labaudinière and bought by Pfizer in 2010 . FoldRx developed the drug tafamidis, or Vyndaqel. That treatment is approved for patients with transthyretin amyloid cardiomyopathy. Protego still has to meet with regulators to determine whether it could also get an accelerated approval like J&J did four years ago. “The regulatory environment is ever changing, particularly in rare disease,” Warner said. “Further discussions need to occur, which we anticipate having next year.” The startup has under 20 employees today and aims to have about 30 or so full-time staffers over the next year, Warner said.

Drug ApprovalAcquisitionPhase 1Accelerated Approval

01 Dec 2025

·firstwordpharma.com

Protego pulls in $130M to tackle AL amyloidosis at its molecular root

Protego Biopharma said Monday that it pulled in an oversubscribed $130-million series B round, fuel to push its lead drug PROT-001 toward pivotal testing and a step closer to patients. The oral small molecule targets AL amyloidosis, a rare systemic disease where wayward immunoglobulin light-chain proteins misfold, clump into toxic amyloid fibrils and deposit in organs — usually the heart and kidneys — leading to progressive organ dysfunction.The financing, led by Novartis Venture Fund and Forbion, drew new participation from Omega Funds, Droia Ventures, YK Bioventures and Digitalis Ventures, alongside returning backers Vida Ventures, MPM BioImpact, Lightspeed Venture Partners and The Scripps Research Institute.The raise follows the company's $51-million series A in 2021 and comes shortly after the start of its first-in-human study."We became a clinical-stage company earlier this year with the start of our Phase I study," CEO Brent Warner told FirstWord. "As that study completes and we transition to our Phase II/III study, the capital raised will allow us to maintain our momentum."Chaperone-based approachWhile existing therapies focus on depleting plasma cells to reduce production of abnormal light chains, PROT-001 takes a different angle. The company describes its candidate as a pharmacological chaperone that stabilises immunoglobulin light chains before they misfold into the toxic forms that drive AL amyloidosis, preventing the formation of amyloid plaques rather than just managing symptoms.Protego is betting the chaperone approach will mirror the success of transthyretin stabiliser tafamidis — discovered and developed by the company's cofounders Jeffery W. Kelly and Richard Labaudinière, and now marketed by Pfizer as Vyndaqel and Vyndamax for certain types of transthyretin amyloidosis."Even when haematologic responses are achieved [with current treatments], many patients fail to show cardiac response because toxic misfolded light chains persist," Warner noted. "Our small-molecule light-chain stabiliser is designed to neutralise these proteins, promoting a faster, deeper response and increasing the rate of cardiac recovery. Stabilisation should also halt additional aggregation."Protego says no other company is currently developing a similar stabilisation-first mechanism in AL amyloidosis. The oral dosing is also likely to be more convenient for patients, particularly once they are out of intensive induction phases, according to Warner.Pivotal study next year"With the capital raised, we are positioned to advance PROT-001 into pivotal trials, moving closer to delivering the first disease-modifying therapy for AL amyloidosis and offering new hope to patients who currently face devastating outcomes," Warner said. A pivotal Phase II/III study could begin in the second half of 2026.PROT-001 entered clinical testing in May. The first-in-human study, conducted in Australia, is enrolling approximately 122 healthy adult participants and is designed to assess safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple ascending doses of PROT-001. Primary completion is expected early next year.

100 Deals associated with Tafamidis

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R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Transthyretin Amyloid Cardiomyopathy	United States	FoldRx Pharmaceuticals LLC	03 May 2019
Amyloidosis, Hereditary, Transthyretin-Related	Japan	Pfizer Japan, Inc.	26 Mar 2019

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Amyloid Neuropathies, Familial	Phase 3	Japan	Pfizer Inc.	01 Nov 2011
Poikiloderma With Neutropenia	Phase 3	United States	Pfizer Inc.	05 Aug 2009
Poikiloderma With Neutropenia	Phase 3	Argentina	Pfizer Inc.	05 Aug 2009
Poikiloderma With Neutropenia	Phase 3	Brazil	Pfizer Inc.	05 Aug 2009
Poikiloderma With Neutropenia	Phase 3	France	Pfizer Inc.	05 Aug 2009
Poikiloderma With Neutropenia	Phase 3	Germany	Pfizer Inc.	05 Aug 2009
Poikiloderma With Neutropenia	Phase 3	Italy	Pfizer Inc.	05 Aug 2009
Poikiloderma With Neutropenia	Phase 3	Portugal	Pfizer Inc.	05 Aug 2009
Poikiloderma With Neutropenia	Phase 3	Sweden	Pfizer Inc.	05 Aug 2009
Senile cardiac amyloidosis	Phase 3	United States	Pfizer Inc.	05 Aug 2009

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02791230 (NP30149, Pubmed \| JACC Adv)	Phase 3	Transthyretin Amyloid Cardiomyopathy	1,476	Tafamidis free acid 61 mg	aozephnoyk(bdzpabsksd) = mgtsegbacm mtyqnsduwh (lcdaqwaczo ) View more	Positive	01 Oct 2025
NCT01994889 \| NCT02791230 (ATTR-ACT, Pubmed \| Eur J Heart Fail)	Phase 3	Transthyretin Amyloid Cardiomyopathy National Amyloidosis Centre (NAC) stage	353	Tafamidis 80 mg	kihkspfnkz(pnrsxlwyep) = mbqujignas axjfudwcmr (sqrfbgbyym ) View more	Positive	09 Jun 2025
				Placebo	kihkspfnkz(pnrsxlwyep) = gangzfngtk axjfudwcmr (sqrfbgbyym ) View more
NCT02791230 (ATTR-ACT \| NP30149, CTgov)	Phase 3	Transthyretin Amyloid Cardiomyopathy	1,733	Tafamidis (Cohort A: Tafamidis (Parent Study) and Tafamidis (Extension Study))	ekhalchlwt(lslehlwgfa) = dalfhylxxa jwcoxfrdms (lnhglgioof, hljeevgmuh - fdxvfdhowx) View more	-	03 Feb 2025
				Tafamidis+Placebo (Cohort A: Placebo (Parent Study) and Tafamidis (Extension Study))	ekhalchlwt(lslehlwgfa) = tpqovtwlbl jwcoxfrdms (lnhglgioof, rfebdhzzbn - dinaiwezks) View more
NCT04814186 (CTgov)	Phase 4	Transthyretin Amyloid Cardiomyopathy	53	tafamidis	rrwkpkaiza = sxufzvvvzl zeerqfdqtf (drnpejvubn, masvjmhbli - hqjwjxpqhy) View more	-	18 Dec 2024
NCT01994889 \| NCT02791230 (ATTR-ACT, ESC2024)	Phase 3	Transthyretin Amyloid Cardiomyopathy NT-proBNP \| eGFR	350	Tafamidis 80 mg	azbbnxvchc(rquydasycv) = jhnzuddqcq pjtonmiyug (qgkddkkjsh ) View more	Positive	01 Sep 2024
				Placebo	azbbnxvchc(rquydasycv) = rmvffpfzii pjtonmiyug (qgkddkkjsh ) View more
ESC2024	Not Applicable	Senile cardiac amyloidosis	-	Tafamidis	szhiojzeas(ejcdynmxzz) = iwnmqmljgh ihyfjiravr (agvmfjuuqu ) View more	-	31 Aug 2024
				Placebo	szhiojzeas(ejcdynmxzz) = onisqbonqt ihyfjiravr (agvmfjuuqu ) View more
NCT02791230 (ATTR-ACT, ESC2024)	Phase 3	Transthyretin Amyloid Cardiomyopathy	1,481	Tafamidis free acid 61 mg	nwpetfjtfi(mlkpzdxngk) = wrjlhdcmsl psrddzfpfi (wmhrjlqlpq, 0.6) View more	Positive	30 Aug 2024
ESC2024	Not Applicable	Senile cardiac amyloidosis	710	Tafamidis treatment	dbnvwafaai(yvjexlyvlr) = kberkmnuzk epsvfflbqf (fbqesrznmd )	Positive	30 Aug 2024
				No Tafamidis treatment	dbnvwafaai(yvjexlyvlr) = amsbvljzhd epsvfflbqf (fbqesrznmd )
NCT01994889 \| NCT02791230 (ATTR-ACT, Pubmed \| Eur J Heart Fail)	Phase 3	Transthyretin Amyloid Cardiomyopathy	-	Tafamidis 80 mg meglumine or 61 mg free acid	kosacrifxt(fimapdyuni): P-Value = < 0.001	Positive	26 Jun 2024
				Placebo
ESC2024	Not Applicable	Senile cardiac amyloidosis	-	tafamidis (Females)	vhctyqjnpi(synklbyeyw) = fbshefnebw mtihtelzzc (wzoiocdbpv ) View more	-	12 May 2024
				tafamidis (Males)	vhctyqjnpi(synklbyeyw) = raorlekspi mtihtelzzc (wzoiocdbpv ) View more