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
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
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
Sources: https://www.scripps.edu/newsandviews/e_20120604/tafamidis.html https://www.ncbi.nlm.nih.gov/pubmed/26894299
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
Primary Target | Pharmacology | Condition | Potency |
---|---|---|---|
Target ID: CHEMBL3194 |
2.0 nM [Kd] |
Conditions
Condition | Modality | Targets | Highest Phase | Product |
---|---|---|---|---|
Primary | Vyndaqel Approved UseVyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage 1
symptomatic polyneuropathy to delay peripheral neurologic impairment. Launch Date2011 |
Cmax
Value | Dose | Co-administered | Analyte | Population |
---|---|---|---|---|
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
Value | Dose | Co-administered | Analyte | Population |
---|---|---|---|---|
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
Value | Dose | Co-administered | Analyte | Population |
---|---|---|---|---|
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
Value | Dose | Co-administered | Analyte | Population |
---|---|---|---|---|
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
Dose | Population | Adverse 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... Other AEs: Hordeolum (grade 1) 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 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%) Sources: 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%) |
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%) Sources: 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%) |
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%) Sources: 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%) |
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%) Sources: Liver function test increased (3.4%) |
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: |
Other AEs: Diarrhea, Upper abdominal pain... Other AEs: Diarrhea (11.4%) Sources: 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%) |
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) Sources: Vaginal infection (grade 1-2, >10) Diarrhoea (grade 1-2, >10) Upper abdominal pain (grade 1-2, >10) |
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%) Sources: Pneumonia (14.4%) Falling (31.1%) Lens disorder (9.5%) Hepatotoxicity (23.1%) Dysfunction thyroid (8.3%) Asthenic conditions (25%) |
AEs
AE | Significance | Dose | Population |
---|---|---|---|
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
CYP3A4 | CYP2C9 | CYP2D6 | hERG |
---|---|---|---|
OverviewOther
Other Inhibitor | Other Substrate | Other Inducer |
---|---|---|
Drug as perpetrator
Target | Modality | Activity | Metabolite | Clinical 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 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/211996Orig1s000,%20212161Orig1s000PharmR.pdf#page=28 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% Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/211996Orig1s000,%20212161Orig1s000ClinPharmR.pdf#page=18 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% Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/211996Orig1s000,%20212161Orig1s000ClinPharmR.pdf#page=18 Page: 18.0 |
|||
yes [IC50 1.16 uM] | ||||
yes [IC50 2.36 uM] | ||||
yes [IC50 30 uM] | ||||
yes |
Drug as victim
Target | Modality | Activity | Metabolite | Clinical evidence |
---|---|---|---|---|
major | ||||
major | ||||
major | ||||
minor | ||||
minor | ||||
minor | ||||
minor | ||||
no | ||||
no | ||||
no | ||||
no | ||||
no | ||||
no | ||||
no | ||||
no | ||||
no |
Tox targets
Target | Modality | Activity | Metabolite | Clinical evidence |
---|---|---|---|---|
PubMed
Title | Date | PubMed |
---|---|---|
Fibril in senile systemic amyloidosis is derived from normal transthyretin. | 1990 Apr |
|
Familial amyloid polyneuropathy: new developments in genetics and treatment. | 1996 Oct |
|
Variant-sequence transthyretin (isoleucine 122) in late-onset cardiac amyloidosis in black Americans. | 1997 Feb 13 |
|
Evidence for direct binding of fatty acids and eicosanoids to human peroxisome proliferators-activated receptor alpha. | 1999 Jul 14 |
|
AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin. | 2013 Jun 11 |
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).
Name | Type | Language | ||
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Official Name | English | ||
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Common Name | English | ||
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Common Name | English | ||
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Common Name | English | ||
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Brand Name | English | ||
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Common Name | English | ||
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Common Name | English | ||
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Code | English | ||
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Common Name | English |
Classification Tree | Code System | Code | ||
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FDA ORPHAN DRUG |
363312
Created by
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NCI_THESAURUS |
C87006
Created by
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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
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Code System | Code | Type | Description | ||
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DTXSID50915094
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PRIMARY | |||
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CHEMBL2103837
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C84194
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PRIMARY | |||
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100000124128
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ZU7CF08A1A
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PRIMARY | |||
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DB05352
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1727003
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PRIMARY | RxNorm | ||
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24970412
Created by
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PRIMARY | |||
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UU-58
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PRIMARY | |||
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SUB31457
Created by
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VYNDAQEL
Created by
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PRIMARY | APPROVED SEPTEMBER 2013 | ||
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79345
Created by
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PRIMARY | |||
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ZU7CF08A1A
Created by
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PRIMARY | |||
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951395-08-7
Created by
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PRIMARY | |||
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m11717
Created by
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PRIMARY |
ACTIVE MOIETY
PARENT (SALT/SOLVATE)
SUBSTANCE RECORD