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Details

Stereochemistry ABSOLUTE
Molecular Formula C14H7Cl2NO3.C7H17NO5
Molecular Weight 503.33
Optical Activity UNSPECIFIED
Defined Stereocenters 4 / 4
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of TAFAMIDIS MEGLUMINE

SMILES

CNC[C@H](O)[C@@H](O)[C@H](O)[C@H](O)CO.OC(=O)C1=CC=C2N=C(OC2=C1)C3=CC(Cl)=CC(Cl)=C3

InChI

InChIKey=DQJDBUPLRMRBAB-WZTVWXICSA-N
InChI=1S/C14H7Cl2NO3.C7H17NO5/c15-9-3-8(4-10(16)6-9)13-17-11-2-1-7(14(18)19)5-12(11)20-13;1-8-2-4(10)6(12)7(13)5(11)3-9/h1-6H,(H,18,19);4-13H,2-3H2,1H3/t;4-,5+,6+,7+/m.0/s1

HIDE SMILES / InChI

Molecular Formula C7H17NO5
Molecular Weight 195.2136
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 4 / 4
E/Z Centers 0
Optical Activity UNSPECIFIED

Molecular Formula C14H7Cl2NO3
Molecular Weight 308.116
Charge 0
Count
Stereochemistry ACHIRAL
Additional Stereochemistry No
Defined Stereocenters 0 / 0
E/Z Centers 0
Optical Activity NONE

Tafamidis meglumine (Vyndaqel®, Pfizer) is a novel, first-in-class drug for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP), a rare neurodegenerative disorder characterized by progressive sensory, motor and autonomic impairment that is ultimately fatal. Pathogenic mutations in the transthyretin (TTR) protein lead to destabilization of its tetrameric structure and subsequent formation of amyloid aggregates. Tafamidis is a small-molecule inhibitor that binds selectively to TTR in human plasma and kinetically stabilizes the tetrameric structure of both wild-type TTR and a number of different mutants. Clinical trials indicate that tafamidis slows disease progression in patients with TTR-FAP and reduces the burden of disease, demonstrating improvement in small and large nerve fiber function, modified body mass index and lower extremity neurological examination. Tafamidis meglumine has been launched for TTR FAP in the EU, Japan, Argentina, Malta and Mexico, and is preregistration in the US for this indication.

Originator

Curator's Comment: Tafamidis was discovered in the Jeffery W. Kelly Laboratory at The Scripps Research Institute using a structure-based drug design strategy and was developed at FoldRx pharmaceuticals.

Approval Year

Targets

Targets

Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Vyndaqel

Approved Use

Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment.

Launch Date

2011
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
11.99 μg/mL
80 mg 1 times / day steady-state, oral
dose: 80 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
TAFAMIDIS MEGLUMINE plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
3 μg/mL
20 mg 1 times / day steady-state, oral
dose: 20 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
TAFAMIDIS MEGLUMINE plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
241.5 μg × h/mL
80 mg 1 times / day steady-state, oral
dose: 80 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
TAFAMIDIS MEGLUMINE plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
60.39 μg × h/mL
20 mg 1 times / day steady-state, oral
dose: 20 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
TAFAMIDIS MEGLUMINE plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
57 h
80 mg 1 times / day steady-state, oral
dose: 80 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
TAFAMIDIS MEGLUMINE plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
57 h
20 mg 1 times / day steady-state, oral
dose: 20 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
TAFAMIDIS MEGLUMINE plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
Funbound

Funbound

ValueDoseCo-administeredAnalytePopulation
1%
80 mg 1 times / day steady-state, oral
dose: 80 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
TAFAMIDIS MEGLUMINE plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
1%
20 mg 1 times / day steady-state, oral
dose: 20 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
TAFAMIDIS MEGLUMINE plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
Doses

Doses

DosePopulationAdverse events​
480 mg 1 times / day single, oral
Highest studied dose
Dose: 480 mg, 1 times / day
Route: oral
Route: single
Dose: 480 mg, 1 times / day
Sources:
healthy, 24 to 54 years
n = 9
Health Status: healthy
Age Group: 24 to 54 years
Sex: M
Population Size: 9
Sources:
Other AEs: Hordeolum...
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Disc. AE: Urinary tract infection...
Other AEs: Neuralgia, Diarrhea...
AEs leading to
discontinuation/dose reduction:
Urinary tract infection (3.1%)
Other AEs:
Neuralgia (3.1%)
Diarrhea (26.2%)
Urinary tract infection (23.1%)
Pain in extremity (16.9%)
Influenza (15.4%)
Headache (15.4%)
Nasopharyngitis (13.8%)
Upper abdominal pain (12.3%)
Nausea (12.3%)
Vomiting (10.8%)
Lacrimation decreased (9.2%)
Myalgia (7.7%)
Punctate keratitis (7.7%)
Back pain (7.7%)
Vaginal infection (6.2%)
Peripheral edema (6.2%)
Constipation (6.2%)
Pharyngitis (6.2%)
Upper respiratory tract infection (6.2%)
Thermal burn (6.2%)
Anxiety (6.2%)
Depression (6.2%)
Erectile dysfunction (6.2%)
Paresthesia (4.6%)
Abdominal pain (4.6%)
Weight decreased (4.6%)
Vertigo (4.6%)
Pharyngolaryngeal pain (3.1%)
Muscle spasms (3.1%)
AV block first degree (3.1%)
Dizziness (3.1%)
Hypoesthesia (1.5%)
Sources:
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Disc. AE: Transient ischemic attack...
Other AEs: Fall, Diarrhea...
AEs leading to
discontinuation/dose reduction:
Transient ischemic attack (grade 1, 4.8%)
Other AEs:
Fall (24%)
Diarrhea (24%)
Pain in extremity (19%)
Dizziness (14%)
Dyspnea (14%)
Vomiting (14%)
Constipation (14%)
Ankle fracture (4.8%)
Malaise (4.8%)
Urinary retention (4.8%)
Sources:
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Other AEs: Diarrhea, Upper abdominal pain...
Other AEs:
Diarrhea (14.2%)
Upper abdominal pain (8%)
Urinary tract infection (12.5%)
Hypersensitivity (14.8%)
Hepatotoxicity (21.6%)
Dysfunction thyroid (9.1%)
Cardiac arrhythmia (37.5%)
Hypotension orthostatic (42%)
Infection (65.9%)
Pancreatitis (0.6%)
Pneumonia (15.3%)
Coordination and balance disturbances (9.1%)
Balance disorder (8.5%)
Fall (24.4%)
Accidental injury (35.2%)
Falling (30%)
Lens disorder (10.2%)
Hepatotoxicity (21.6%)
Dysfunction thyroid (9.1%)
Asthenic conditions (25.6%)
Retinol binding protein increased (44%)
AST increased (7.39%)
ALP increased (6.25%)
Bilirubin total increased (0.57%)
Sources:
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Other AEs: Flatulence, Liver function test increased...
Other AEs:
Flatulence (4.5%)
Liver function test increased (3.4%)
Sources:
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Other AEs: Diarrhea, Upper abdominal pain...
Other AEs:
Diarrhea (11.4%)
Upper abdominal pain (9.1%)
Urinary tract infection (11.4%)
Hypersensitivity (10.2%)
Hepatotoxicity (26.1%)
Dysfunction thyroid (6.8%)
Cardiac arrhythmia (33%)
Hypotension orthostatic (48.9%)
Infection (55.7%)
Pneumonia (12.5%)
Coordination and balance disturbances (2.3%)
Balance disorder (2.3%)
Fall (30.7%)
Accidental injury (42%)
Falling (33%)
Lens disorder (8%)
Hepatotoxicity (26.1%)
Dysfunction thyroid (6.8%)
Asthenic conditions (23.9%)
Retinol binding protein increased (51%)
AST increased (3.41%)
ALP increased (5.68%)
Bilirubin total increased (1.14%)
Sources:
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 127
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 127
Sources:
Other AEs: Urinary tract infection, Vaginal infection...
Other AEs:
Urinary tract infection (grade 1-2, >10)
Vaginal infection (grade 1-2, >10)
Diarrhoea (grade 1-2, >10)
Upper abdominal pain (grade 1-2, >10)
Sources:
50 mg 1 times / day multiple, oral (mean)
Dose: 50 mg, 1 times / day
Route: oral
Route: multiple
Dose: 50 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 264
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 264
Sources:
Other AEs: Infection, Pneumonia...
Other AEs:
Infection (62.5%)
Pneumonia (14.4%)
Falling (31.1%)
Lens disorder (9.5%)
Hepatotoxicity (23.1%)
Dysfunction thyroid (8.3%)
Asthenic conditions (25%)
Sources:
AEs

AEs

AESignificanceDosePopulation
Hordeolum grade 1
480 mg 1 times / day single, oral
Highest studied dose
Dose: 480 mg, 1 times / day
Route: oral
Route: single
Dose: 480 mg, 1 times / day
Sources:
healthy, 24 to 54 years
n = 9
Health Status: healthy
Age Group: 24 to 54 years
Sex: M
Population Size: 9
Sources:
Hypoesthesia 1.5%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Vomiting 10.8%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Nausea 12.3%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Upper abdominal pain 12.3%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Nasopharyngitis 13.8%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Headache 15.4%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Influenza 15.4%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Pain in extremity 16.9%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Urinary tract infection 23.1%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Diarrhea 26.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
AV block first degree 3.1%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Dizziness 3.1%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Muscle spasms 3.1%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Neuralgia 3.1%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Pharyngolaryngeal pain 3.1%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Urinary tract infection 3.1%
Disc. AE
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Abdominal pain 4.6%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Paresthesia 4.6%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Vertigo 4.6%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Weight decreased 4.6%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Anxiety 6.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Constipation 6.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Depression 6.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Erectile dysfunction 6.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Peripheral edema 6.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Pharyngitis 6.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Thermal burn 6.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Upper respiratory tract infection 6.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Vaginal infection 6.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Back pain 7.7%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Myalgia 7.7%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Punctate keratitis 7.7%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Lacrimation decreased 9.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 39.8 years
n = 65
Health Status: unhealthy
Condition: transthyretin familial amyloid polyneuropathy
Age Group: mean 39.8 years
Sex: M+F
Population Size: 65
Sources:
Constipation 14%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Dizziness 14%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Dyspnea 14%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Vomiting 14%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Pain in extremity 19%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Diarrhea 24%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Fall 24%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Ankle fracture 4.8%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Malaise 4.8%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Urinary retention 4.8%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Transient ischemic attack grade 1, 4.8%
Disc. AE
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, mean 63.1 years
n = 21
Health Status: unhealthy
Condition: non-Val30Met transthyretin amyloidosis
Age Group: mean 63.1 years
Sex: M+F
Population Size: 21
Sources:
Bilirubin total increased 0.57%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Pancreatitis 0.6%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Lens disorder 10.2%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Urinary tract infection 12.5%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Diarrhea 14.2%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Hypersensitivity 14.8%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Pneumonia 15.3%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Hepatotoxicity 21.6%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Hepatotoxicity 21.6%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Fall 24.4%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Asthenic conditions 25.6%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Falling 30%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Accidental injury 35.2%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Cardiac arrhythmia 37.5%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Hypotension orthostatic 42%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Retinol binding protein increased 44%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
ALP increased 6.25%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Infection 65.9%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
AST increased 7.39%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Upper abdominal pain 8%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Balance disorder 8.5%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Coordination and balance disturbances 9.1%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Dysfunction thyroid 9.1%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Dysfunction thyroid 9.1%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Liver function test increased 3.4%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Flatulence 4.5%
80 mg 1 times / day multiple, oral
Recommended
Dose: 80 mg, 1 times / day
Route: oral
Route: multiple
Dose: 80 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 176
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 176
Sources:
Bilirubin total increased 1.14%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Hypersensitivity 10.2%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Diarrhea 11.4%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Urinary tract infection 11.4%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Pneumonia 12.5%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Balance disorder 2.3%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Coordination and balance disturbances 2.3%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Asthenic conditions 23.9%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Hepatotoxicity 26.1%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Hepatotoxicity 26.1%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
AST increased 3.41%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Fall 30.7%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Cardiac arrhythmia 33%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Falling 33%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Accidental injury 42%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Hypotension orthostatic 48.9%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
ALP increased 5.68%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Retinol binding protein increased 51%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Infection 55.7%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Dysfunction thyroid 6.8%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Dysfunction thyroid 6.8%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Lens disorder 8%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Upper abdominal pain 9.1%
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 88
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 88
Sources:
Diarrhoea grade 1-2, >10
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 127
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 127
Sources:
Upper abdominal pain grade 1-2, >10
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 127
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 127
Sources:
Urinary tract infection grade 1-2, >10
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 127
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 127
Sources:
Vaginal infection grade 1-2, >10
20 mg 1 times / day multiple, oral
Dose: 20 mg, 1 times / day
Route: oral
Route: multiple
Dose: 20 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 127
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 127
Sources:
Pneumonia 14.4%
50 mg 1 times / day multiple, oral (mean)
Dose: 50 mg, 1 times / day
Route: oral
Route: multiple
Dose: 50 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 264
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 264
Sources:
Hepatotoxicity 23.1%
50 mg 1 times / day multiple, oral (mean)
Dose: 50 mg, 1 times / day
Route: oral
Route: multiple
Dose: 50 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 264
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 264
Sources:
Asthenic conditions 25%
50 mg 1 times / day multiple, oral (mean)
Dose: 50 mg, 1 times / day
Route: oral
Route: multiple
Dose: 50 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 264
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 264
Sources:
Falling 31.1%
50 mg 1 times / day multiple, oral (mean)
Dose: 50 mg, 1 times / day
Route: oral
Route: multiple
Dose: 50 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 264
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 264
Sources:
Infection 62.5%
50 mg 1 times / day multiple, oral (mean)
Dose: 50 mg, 1 times / day
Route: oral
Route: multiple
Dose: 50 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 264
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 264
Sources:
Dysfunction thyroid 8.3%
50 mg 1 times / day multiple, oral (mean)
Dose: 50 mg, 1 times / day
Route: oral
Route: multiple
Dose: 50 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 264
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 264
Sources:
Lens disorder 9.5%
50 mg 1 times / day multiple, oral (mean)
Dose: 50 mg, 1 times / day
Route: oral
Route: multiple
Dose: 50 mg, 1 times / day
Sources:
unhealthy, median age 75 years
n = 264
Health Status: unhealthy
Condition: transthyretin mediated amyloidosis
Age Group: median age 75 years
Sex: M+F
Population Size: 264
Sources:
Overview

Overview

Drug as perpetrator​

Drug as perpetrator​

TargetModalityActivityMetaboliteClinical evidence
moderate [Ki 19 uM]
no [IC50 83 uM]
no [IC50 86 uM]
no [IC50 >100 uM]
no [IC50 >100 uM]
no
no
no
no
no
no
no
no
weak [Ki >100 uM]
weak [Ki >100 uM]
weak [Ki >100 uM]
weak [Ki >100 uM]
weak [Ki >100 uM]
no (co-administration study)
Comment: clinical DDI study with midazolam, a probe CYP3A substrate, did not show any clinically significant impact on the PK of midazolam and its active metabolite when co-administered
Page: 28.0
yes [EC50 25 uM]
unlikely
Comment: maximal decrease in AUC of CYP2B6 substrates using static-mechanistic model was predicted to be 15%
Page: 18.0
yes [EC50 28 uM]
unlikely (co-administration study)
Comment: co-administration wtih midazolam did not show any clinically significant impact on PK of midazolam; maximal decrease in AUC of CYP3A4 substrates using static-mechanistic model was predicted to be 9%
Page: 18.0
yes [IC50 1.16 uM]
yes [IC50 2.36 uM]
yes [IC50 30 uM]
yes
Drug as victim

Drug as victim

TargetModalityActivityMetaboliteClinical evidence
major
major
major
minor
minor
minor
minor
no
no
no
no
no
no
no
no
no
Tox targets

Tox targets

PubMed

PubMed

TitleDatePubMed
A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves.
1952 Sep
Fibril in senile systemic amyloidosis is derived from normal transthyretin.
1990 Apr
Familial amyloid polyneuropathy: new developments in genetics and treatment.
1996 Oct
Patents

Sample Use Guides

The recommended dose of tafamidis meglumine is 20 mg orally once daily.
Route of Administration: Oral
Tafamidis effectively inhibited fibril formation induced by acid denaturation in a concentration-dependent manner with similar potency for wild-type TTR, V30M TTR and V122I TTR, reaching its EC50 at a tafamidis:TTR stoichiometry of <1 (EC50 was in the range of 2.7-3.2 uM, corresponding to a tafamidis: TTR stoichiometry range of 0.75-0.9).
Substance Class Chemical
Created
by admin
on Fri Dec 15 16:13:49 GMT 2023
Edited
by admin
on Fri Dec 15 16:13:49 GMT 2023
Record UNII
ZU7CF08A1A
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
TAFAMIDIS MEGLUMINE
DASH   USAN   WHO-DD  
USAN  
Official Name English
TAFAMIDIS MEGLUMINE [MI]
Common Name English
TAFAMIDIS MEGLUMINE [ORANGE BOOK]
Common Name English
D-GLUCO-2,3,4,5,6-PENTAHYDROXY-N-METHYLHEXAN-1-AMINIUM 2-(3,5-DICHLOROPHENYL)BENZOXAZOLE-6-CARBOXYLATE
Common Name English
VYNDAQEL
Brand Name English
TAFAMIDIS MEGLUMINE [USAN]
Common Name English
Tafamidis meglumine [WHO-DD]
Common Name English
TAFAMIDIS MEGLUMINE [JAN]
Common Name English
FX-1006A
Code English
D-GLUCITOL, 1-DEOXY-1-(METHYLAMINO)-, 2-(3,5-DICHLOROPHENYL)-6-BENZOXAZOLECARBOXYLATE (1:1)
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 363312
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
NCI_THESAURUS C87006
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
EU-Orphan Drug EU/3/06/401
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
Code System Code Type Description
EPA CompTox
DTXSID50915094
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
ChEMBL
CHEMBL2103837
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
NCI_THESAURUS
C84194
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
SMS_ID
100000124128
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
DAILYMED
ZU7CF08A1A
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
DRUG BANK
DB05352
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
RXCUI
1727003
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY RxNorm
PUBCHEM
24970412
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
USAN
UU-58
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
EVMPD
SUB31457
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
JAPANESE REVIEW
VYNDAQEL
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY APPROVED SEPTEMBER 2013
CHEBI
79345
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
FDA UNII
ZU7CF08A1A
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
CAS
951395-08-7
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
MERCK INDEX
m11717
Created by admin on Fri Dec 15 16:13:49 GMT 2023 , Edited by admin on Fri Dec 15 16:13:49 GMT 2023
PRIMARY
Related Record Type Details
PARENT -> SALT/SOLVATE
PARENT -> SALT/SOLVATE
Related Record Type Details
ACTIVE MOIETY