Tafamidis
/api/v1/drug/tafamidisMechanism of action
Sourced from openFDATafamidis is a selective stabilizer of TTR. Tafamidis binds to TTR at the thyroxine binding sites, stabilizing the tetramer and slowing dissociation into monomers, the rate-limiting step in the amyloidogenic process.
Indications
Sourced from openFDA- 1. INDICATIONS AND USAGE VYNDAQEL and VYNDAMAX are indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.
Contraindications
Sourced from openFDA- 4. CONTRAINDICATIONS None.contraindicated
Dosage & administration
Sourced from openFDA2. DOSAGE AND ADMINISTRATION The recommended dosage is either: • VYNDAQEL 80 mg orally once daily, or • VYNDAMAX 61 mg orally once daily ( 2.1 ) • VYNDAMAX and VYNDAQEL are not substitutable on a per mg basis. ( 2.1 ) 2.1 Recommended Dosage The recommended dosage is either VYNDAQEL 80 mg (four 20-mg tafamidis meglumine capsules) orally once daily or VYNDAMAX 61 mg (one 61-mg tafamidis capsule) orally once daily. VYNDAMAX and VYNDAQEL are not substitutable on a per mg basis [see Clinical Pharmacology (12.3) ] . 2.2 Administration Instructions The capsules should be swallowed whole and not crushed or cut. If a dose is missed, instruct patients to take the dose as soon as remembered or to skip the missed dose and take the next dose at the regularly scheduled time. Do not double the dose.
Adverse reactions
Sourced from openFDA6. ADVERSE REACTIONS To report SUSPECTED ADVERSE REACTIONS, contact Pfizer Inc. at 1-800-438-1985 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The data reflect exposure of 377 ATTR-CM patients to 20 mg or 80 mg (administered as four 20-mg capsules) of VYNDAQEL administered daily for an average of 24.5 months (ranging from 1 day to 111 months). Adverse events were assessed from ATTR-CM clinical trials with VYNDAQEL, primarily a 30-month placebo-controlled trial [see Clinical Studies (14) ] . The frequency of adverse events in patients treated with VYNDAQEL 20 mg (n=88) or 80 mg (n=176; administered as four 20-mg capsules) was similar to that with placebo (n=177). In the 30-month placebo-controlled trial, similar proportions of VYNDAQEL-treated patients and placebo-treated patients discontinued the study drug because of an adverse event: 12 (7%), 5 (6%), and 11 (6%) from the VYNDAQEL 80-mg, VYNDAQEL 20-mg, and placebo groups, respectively.
Use in specific populations
Sourced from openFDA8. USE IN SPECIFIC POPULATIONS • Pregnancy: Based on animal studies, may cause fetal harm. ( 8.1 ) • Lactation: Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on findings from animal studies, VYNDAQEL and VYNDAMAX may cause fetal harm when administered to a pregnant woman. However, limited available human data with VYNDAQEL use in pregnant women (at a dose of 20 mg per day) have not identified any drug-associated risks for major birth defects, miscarriage, or adverse maternal or fetal outcomes. In animal reproductive studies, oral administration of tafamidis meglumine to pregnant rabbits during organogenesis resulted in adverse effects on development (embryofetal mortality, fetal body weight reduction and fetal malformation) at a dosage providing approximately 9 times the human exposure (AUC) at the maximum recommended human dose (MRHD) of VYNDAQEL (80 mg), and increased incidence of fetal skeletal variation at a dosage providing equivalent human exposure (AUC) at the MRHD. Postnatal mortality, growth retardation, and impaired learning and memory were observed in offspring of pregnant rats administered tafamidis meglumine during gestation and lactation at a dosage approximately 2 times the MRHD based on body surface area (mg/m 2 ) (see Data ) . Advise pregnant women of the potential risk to a fetus.
Pharmacokinetics
Sourced from openFDA- Metabolism
- No clinically significant differences in steady state C max and area under the plasma concentration over time curve (AUC) of tafamidis were observed for VYNDAMAX 61-mg capsule compared to VYNDAQEL administered as four 20-mg capsules. Tafamidis exposure increases proportionally over single (up to 480 mg) or multiple (up to 80 mg) (1 to 6 times the approved recommended dosage) once daily dosing.
Overdosage
Sourced from openFDA10. OVERDOSAGE There is minimal clinical experience with overdose. During clinical trials, two patients accidentally ingested a single VYNDAQEL dose of 160 mg without adverse events. The highest dose of tafamidis meglumine given to healthy volunteers in a clinical trial was 480 mg as a single dose. There was one reported adverse event of mild hordeolum at this dose.
Approval history
Sourced from openFDA- May 3, 2019NDANDA211996Foldrx Pharms
- May 3, 2019NDANDA212161Foldrx Pharms
FAERS reports
- 1Death4,19927%
- 2Cardiac Failure1,0746.9%
- 3Off Label Use1,0486.7%
- 4Dyspnoea9496.1%
- 5Fatigue7034.5%
- 6Fall5703.7%
- 7Atrial Fibrillation5283.4%
- 8Dizziness4582.9%
- 9Asthenia4352.8%
- 10Malaise4212.7%
- 11Condition Aggravated4122.6%
- 12Weight Decreased3822.4%
- 13Cardiac Failure Congestive3642.3%
- 14Disease Progression3482.2%
- 15Hypoacusis3382.2%
Clinical trials
The 10 most recently updated of 56 ClinicalTrials.gov registrations naming Tafamidis as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.
- A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to AcoramidisRecruiting · Phase 4 · Interventional · 50 enrolled · BayerNCT07298044updated 2026-06-08
- Safety, Efficacy, and Pharmacokinetics of Tafamidis in Patients With Transthyretin-mediated Amyloidosis Post Orthotopic Heart TransplantationActive not recruiting · Phase 4 · Interventional · 25 enrolled · University of Texas Southwestern Medical CenterNCT05489523updated 2026-06-04
- A Study to Compare the Amount of Two Forms of Tafamidis in the Blood of Healthy Adults Under Fasting ConditionsActive not recruiting · Phase 1 · Interventional · 15 enrolled · PfizerNCT07587697updated 2026-06-03
- A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in GermanyActive not recruiting · Observational · 1 enrolled · PfizerNCT07494656updated 2026-05-22
- Vyndaqel Capsules Special Investigation (ATTR-CM)Completed · Observational · 1,214 enrolled · PfizerNCT04108091updated 2026-05-18
- Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid CardiomyopathyCompleted · Observational · 50 enrolled · PfizerNCT06393465updated 2026-04-29
- A Study to Compare the Amount of Two Forms of Tafamidis in the Blood of Healthy Adults Under Fed ConditionsRecruiting · Phase 1 · Interventional · 24 enrolled · PfizerNCT07470866updated 2026-04-24
- NatiOnal Referral cenTEr Study of Transthyretin Amyloid Cardiomyopathy(ATTR) Patients on TafamidisRecruiting · Observational · 200 enrolled · Paolo MilaniNCT06251778updated 2026-04-16
- Korean Post-marketing Surveillance Vyndamax® Capsules for the Treatment of Transthyretin Amyloid CardiomyopathyActive not recruiting · Observational · 110 enrolled · PfizerNCT04801329updated 2026-03-30
- Monitoring of Early Disease Progression in Hereditary Transthyretin AmyloidosisCompleted · Observational · 37 enrolled · The Cleveland ClinicNCT03431896updated 2026-03-12
Frequently asked questions
- How does Tafamidis work?
- Tafamidis is a selective stabilizer of TTR. Tafamidis binds to TTR at the thyroxine binding sites, stabilizing the tetramer and slowing dissociation into monomers, the rate-limiting step in the amyloidogenic process.
- What is Tafamidis used for?
- According to FDA labeling, Tafamidis carries indications including: 1. INDICATIONS AND USAGE VYNDAQEL and VYNDAMAX are indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.
- What class of drug is Tafamidis?
- Tafamidis is classified as Other nervous system drugs.
- What are the brand names for Tafamidis?
- Tafamidis is marketed under brand names including Vyndamax, Vyndaqel.
- What are the contraindications for Tafamidis?
- Tafamidis labeling lists contraindications including: 4. CONTRAINDICATIONS None.. Always consult the full prescribing information and a clinician.
tafamidis is illustrative MVP content compiled from public sources. pharmacopeia is for educational and informational use only and is not a substitute for professional medical advice.