Drugs / Tafamidis
Tafamidis
also known as
FX006 · Pf-06291826 · tafamidis free acid · tafamidis meglumine · PF-06291826-00 · PF-06291826-83 · PF-6291826 · tafamidis (Vyndaqel) · +1 more
targets TTR
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed ATTRV122I amyloidosis 2 milestones | |||||
| Filed | US (FDA) | — | ATTRV122I amyloidosis | 2019-01-14 | “the US Food and Drug Administration (FDA) accepted for filing the company’s New Drug Applications (NDAs) for tafamidis for the treatment of transthyretin amyloid cardiomyopathy” pfizer.com ↗ |
| Filed | Japan (PMDA) | — | ATTRV122I amyloidosis | — | “a regulatory marketing application for tafamidis for ATTR-CM was submitted to the Pharmaceuticals and Medical Devices Agency (PMDA) in November 2018” pfizer.com ↗ |
| Rejected familial amyloid neuropathy 1 milestone | |||||
| Rejected (CRL) | US (FDA) | — | familial amyloid neuropathy | — | “Pfizer received a complete response letter from the US Food and Drug Administration (FDA) on its application to approve tafamidis for TTR-FAP in 2012” pfizer.com ↗ “Pfizer received a complete response letter from the FDA on its application to approve tafamidis for TTR-FAP in 2012” pfizer.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | US (FDA) | VYNDAMAX | — | 2019-05-03 | fda.gov ↗ |
| Approved | EU (EMA) | Vyndaqel | — | 2011-11-16 | europa.eu ↗ |
Trials 40
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 118 trials · 2 met primary | ||||||
| Phase 1 | NCT07587697 | May → Aug 2026 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT07470866 | Mar → May 2026 | — | Pfizer | Terminated | No outcome recorded Stop: Business |
| Phase 1 | NCT06705569 | Dec 2024 → Apr 2025 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT06273839 | Feb → May 2024 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT05498701 | Sep → Dec 2022 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT05482308 | Aug → Oct 2022 | — | Pfizer | Completed | Met primary |
| Phase 1 | NCT04253353 | Feb → Aug 2020 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT03662191 | Sep 2018 → Jan 2019 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT03280173 | Sep 2017 → Feb 2018 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT03266705 | Sep 2017 → Feb 2018 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT02746926 | Apr → Jul 2016 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT02697864 | Apr → Jun 2016 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT02534740 | Sep → Dec 2015 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT02406560 | Apr → Jun 2015 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT02189330 | May → Jul 2014 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT01775761 | Jan → Apr 2013 | — | Pfizer | Completed | Met primary |
| Phase 1 | NCT01655511 | Jul → Sep 2012 | — | Pfizer | Completed | No outcome recorded |
| Phase 1 | NCT01369836 | Jul → Aug 2011 | — | Pfizer | Completed | No outcome recorded |
| Phase 211 trials · 2 met primary · 1 missed | ||||||
| Phase 2 | NCT04182672 | Aug 2020 → Dec 2022 | — | The University of Texas Health Science Center, Houston | Completed | No outcome recorded |
| Phase 2 | NCT04160091 | Nov 2019 → Jun 2020 | frozen shoulder, osteoarthritis | Pacira Pharmaceuticals, Inc | Terminated | No outcome recorded |
| Phase 2 | NCT04065074 | Jul → Oct 2019 | osteoarthritis, hip | Pacira Pharmaceuticals, Inc | Completed | No outcome recorded |
| Phase 2 | NCT03382262 | Dec 2017 → Oct 2018 | osteoarthritis, hip | Pacira Pharmaceuticals, Inc | Completed | No outcome recorded |
| Phase 2 | NCT03378076 | Dec 2017 → Mar 2018 | osteoarthritis, knee | Pacira Pharmaceuticals, Inc | Completed | No outcome recorded |
| Phase 2 | NCT02762370 | Apr → Oct 2016 | osteoarthritis, knee, type 2 diabetes mellitus | Pacira Pharmaceuticals, Inc | Completed | Met primary |
| Phase 2 | NCT02468583 | Feb 2015 → Jul 2016 | osteoarthritis, knee | Pacira Pharmaceuticals, Inc | Terminated | No outcome recorded |
| Phase 2 | NCT02003365 | Nov 2013 → Apr 2014 | osteoarthritis, knee | Pacira Pharmaceuticals, Inc | Completed | No outcome recorded |
| Phase 2 | NCT01487200 | Jul → Nov 2012 | osteoarthritis, knee | Pacira Pharmaceuticals, Inc | Completed | No outcome recorded |
| Phase 2 | NCT01487161 | Jun 2012 → Apr 2013 | osteoarthritis, knee | Pacira Pharmaceuticals, Inc | Completed | Missed primary |
| Phase 2 | NCT00630864 | Jun 2008 → Jan 2010 | familial amyloid neuropathy | Pfizer | Completed | Met primary |
| Phase 37 trials · 3 met primary · 1 missed | ||||||
| Phase 3 | NCT03793010 | Dec 2018 → Aug 2019 | osteoarthritis, hip | Pacira Pharmaceuticals, Inc | Terminated | Missed primary |
| Phase 3 | NCT03529942 | Apr 2018 → Nov 2019 | osteoarthritis, knee | Pacira Pharmaceuticals, Inc | Completed | No outcome recorded |
| Phase 3 | NCT02791230 | Jun 2016 → Oct 2023 | amyloidosis | Pfizer | Completed | Met primary |
| Phase 3 | NCT02357459 | Jan 2015 → Jan 2016 | osteoarthritis, knee | Pacira Pharmaceuticals, Inc | Completed | Met primary |
| Phase 3 | NCT01994889 | Dec 2013 → Feb 2018 | amyloidosis | Pfizer | Completed | Met primary |
| Phase 3 | NCT01435655 | Nov 2011 → Feb 2014 | familial amyloid neuropathy | Pfizer | Completed | No outcome recorded |
| Phase 3 | NCT00935012 | Sep 2009 → Nov 2019 | — | Pfizer | Completed | No outcome recorded |
| Phase 44 trials | ||||||
| Phase 4 | NCT06940336 | Jun 2025 → Jan 2028 expected | familial amyloid neuropathy | Qilu Pharmaceutical Co., Ltd. | Recruiting | No outcome recorded |
| Phase 4 | NCT05489523 | May 2023 → May 2027 expected | — | University of Texas Southwestern Medical Center | Active not recruiting | No outcome recorded |
| Phase 4 | NCT04814186 | Jul 2021 → Oct 2023 | amyloidosis | Pfizer | Completed | No outcome recorded |
| Phase 4 | NCT04963985 | Jun 2021 → Jun 2022 | familial amyloid neuropathy | Peking University Third Hospital | Unknown | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 3 | NCT00925002 Comparator | Aug 2009 → Jul 2020 | — | Pfizer | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 12
| Date | Issuer | Release |
|---|---|---|
| 2021-12-20 | Pfizer | Results Pfizer’s VYNDAQEL®/VYNDAMAX® Reduced the Risk of All-Cause Mortality by 41% Among Patients with Transthyretin Amyloid Cardiomyopathy, Five-Year Follow-Up Data Demonstrate pfizer.com ↗
Pfizer Inc. (NYSE: PFE) announced today the publication of a post-hoc, interim analysis showing that treatment with VYNDAQEL ® (tafamidis meglumine) / VYNDAMAX ® (tafamidis) provided a clinically significant survival benefit at five years for patients with transthyretin amyloid cardiomyopathy (ATTR-CM). |
| 2020-11-12 | Pfizer | Results Analysis from Phase 3 ATTR-ACT and Its Long-Term Extension Study Demonstrates VYNDAQEL® 80 mg/VYNDAMAX® 61 mg Significantly Improved Survival in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Compared to VYNDAQEL 20 mg pfizer.com ↗
When adjusting for covariates, including age, biomarkers and functional capacity, the reduction in risk was increased to 43% (p<0.05) for VYNDAQEL 80 mg/VYNDAMAX 61 mg versus VYNDAQEL 20 mg. |
| 2020-02-18 | Pfizer | Regulatory European Commission Approves VYNDAQEL®, the First Treatment in the EU for Transthyretin Amyloid Cardiomyopathy (ATTR-CM) pfizer.com ↗
Pfizer Inc. (NYSE: PFE) announced today that the European Commission (EC) has approved VYNDAQEL ® (tafamidis), a once-daily 61 mg oral capsule, for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). |
| 2019-12-13 | Pfizer | Regulatory Pfizer Receives Positive CHMP Opinion for VYNDAQEL® for Use in Patients with Transthyretin Amyloid Cardiomyopathy, a Rare and Fatal Disease pfizer.com ↗
has adopted a positive opinion recommending the approval of VYNDAQEL® (tafamidis) |
| 2019-05-06 | Pfizer | Regulatory U.S. FDA Approves VYNDAQEL® and VYNDAMAX™ for Use in Patients with Transthyretin Amyloid Cardiomyopathy, a Rare and Fatal Disease pfizer.com ↗
VYNDAQEL and VYNDAMAX are two oral formulations of the first-in-class transthyretin stabilizer tafamidis, and the first and only medicines approved by the FDA to treat ATTR-CM. |
| 2019-01-14 | Pfizer | Regulatory US FDA Accepts Regulatory Submissions for Review of Tafamidis to Treat Transthyretin Amyloid Cardiomyopathy pfizer.com ↗
Pfizer Inc. (NYSE: PFE) announced today that the US Food and Drug Administration (FDA) accepted for filing the company’s New Drug Applications (NDAs) for tafamidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). |
| 2018-09-18 | Pfizer | Results New Sub-group Analyses from the Tafamidis Phase 3 Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) Study Presented at 2018 HFSA Annual Scientific Meeting pfizer.com ↗
A new sensitivity analysis presented today also demonstrated a significant reduction in the combination of all-cause mortality and frequency of all-cause hospitalization compared to placebo over a 30-month period (P=0.0088). |
| 2018-05-23 | Pfizer | Regulatory FDA Grants Breakthrough Therapy Designation for Tafamidis for the Treatment of Patients with Transthyretin Cardiomyopathy pfizer.com ↗
Pfizer Inc. (NYSE:PFE) announced today that tafamidis received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for the treatment of patients with transthyretin cardiomyopathy, a rare, fatal, and underdiagnosed condition associated with progressive heart failure. |
| 2018-03-29 | Pfizer | Results Pfizer Announces Positive Topline Results From Phase 3 ATTR-ACT Study Of Tafamidis In Patients With Transthyretin Cardiomyopathy pfizer.com ↗
The preliminary safety data showed that tafamidis was generally well tolerated in this population and no new safety signals were identified. |
| 2017-08-31 | Pfizer | Results Amyloid Publishes Long-Term Data Analysis from Pfizer Suggesting Tafamidis Delays Progression of TTR-FAP, a Rare Disease pfizer.com ↗
Among all 93 patients in the analysis, tafamidis was generally well-tolerated, with no unexpected safety issues identified. |
| 2017-06-06 | Pfizer | Regulatory Pfizer Receives FDA Fast Track Designation for Tafamidis for Transthyretin Cardiomyopathy pfizer.com ↗
Pfizer Inc. (NYSE:PFE) announced today that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to tafamidis, the company’s investigational treatment for transthyretin cardiomyopathy (TTR-CM). |
| 2016-08-08 | Pfizer | Results Pfizer Announces Publication of New Analysis Showing Long-Term Therapy with VYNDAQEL (tafamidis) Slowed Progression of Rare Neurodegenerative Disease pfizer.com ↗
The published analysis showed that mBMI was preserved during long-term VYNDAQEL treatment, with less than one percent decrease at 5.5 years from baseline. |
All press releases naming this drug 13 releases
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 44 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | tafamidis | “This phase II, open-label, single-treatment arm study evaluated the pharmacodynamics, efficacy, and safety of tafamidis in patients with non-Val30Met transthyretin (TTR) amyloidosis.” PMID 24101373 ↗ Oct 201327“The transthyretin (TTR) stabilizer, tafamidis, has demonstrated efficacy and safety in the treatment of TTR familial amyloid polyneuropathy (20 mg day(-1) ).” PMID 25546001 ↗ Jun 2015 |
| Known as | FX006 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02468583 ↗ |
| Known as | Pf-06291826 | “A Phase 1, Open-Label, Randomized, 2-Cohort, Crossover, Single Dose Study to Estimate the Relative Bioavailability of PF 06291826 (Tafamidis) Following Administration of Two...” NCT02189330 ↗5“A Phase 1, Open-label, Randomized, Crossover, Multiple Dose, Pivotal Bioequivalence Study To Compare Pf-06291826 4 X 20 Mg Tafamidis Meglumine And 61 Mga Tafamidis Free Acid...” NCT03266705 ↗ “A Phase 1, Open-Label, Three-Period Randomized, Single Dose Study To Estimate The Relative Bioavailability Of Pf-06291826 (Tafamidis) Free Acid Capsule Formulation Versus...” NCT02746926 ↗ “A Phase 1, Open-Label, Randomized, Single Dose Study To Estimate The Relative Bioavailability And Food Effect Of PF-06291826 (Tafamidis) Following Administration Of New Soft...” NCT02534740 ↗ “A Study Comparing the Amounts of 2 Different Forms of Tafamidis (PF06291826), Both With and Without Food, In the Blood” NCT03280173 ↗ “DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826) 20 MG OR 80 MG IN COMPARISON TO PLACEBO” NCT01994889 ↗ |
| Known as | PF-06291826-00 | “A PHASE 3 MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE SAFETY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826-83) 20 MG OR 80 MG [OR TAFAMIDIS (PF-06291826-00) 61 MG]...” NCT02791230 ↗ |
| Known as | PF-06291826-83 | “A PHASE 3 MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE SAFETY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826-83) 20 MG OR 80 MG [OR TAFAMIDIS (PF-06291826-00) 61 MG]...” NCT02791230 ↗ |
| Known as | PF-6291826 | “A Study Comparing Amounts of 2 Different Forms of Tafamidis (PF-6291826) in the Blood” NCT03266705 ↗ |
| Known as | tafamidis (Vyndaqel) | “tafamidis (Vyndaqel)” NCT01775761 ↗ |
| Known as | tafamidis free acid | “All patients in the LTE transitioned to tafamidis free acid 61 mg (bioequivalent to tafamidis meglumine 80 mg) following a protocol amendment.” PMID 34923848 ↗ Dec 20211“A new tafamidis free acid tablet is being developed to be bioequivalent to the current commercial 61 mg tafamidis free acid capsule.” NCT06705569 ↗ |
| Known as | Tafamidis meglumin | “Drug: Tafamidis meglumin” NCT01369836 ↗ |
| Known as | tafamidis meglumine | “Early treatment with tafamidis for up to 5.5 years (≥1 dose of tafamidis meglumine 20 mg once daily during the original trial or after switching from placebo in its extension)...” PMID 27494299 ↗ Aug 20161“Patients with transthyretin amyloid cardiomyopathy who completed ATTR-ACT could enroll in an LTE, continuing with the same tafamidis dose or, if previously treated with...” PMID 34923848 ↗ Dec 2021 |
| Route | Oral | “20 mg oral tafamidis” NCT00935012 ↗ |
| Target | TTR | “Tafamidis has been developed as an oral specific stabilizer of transthyretin tetramer.” NCT01435655 ↗ |