Tafamidis

Summary

Tafamidis (CHEMBL2103837) is an approved small-molecule central nervous system drug (ATC N07XX08) targeting TTR; indicated across 4 conditions including cardiomyopathy and amyloidosis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N07XX08
• Targets: 1 (TTR)
• Indications: 4 conditions
• Clinical trials: 27
• Chemistry: 308.1 Da · C14H7Cl2NO3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2103837
Name	Tafamidis
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	11001318
ChEBI	CHEBI:78538
ATC	N07XX08
Molecular formula	C14H7Cl2NO3
Molecular weight	308.1
InChIKey	TXEIIPDJKFWEEC-UHFFFAOYSA-N

SMILES: C1=CC2=C(C=C1C(=O)O)OC(=N2)C3=CC(=CC(=C3)Cl)Cl

IUPAC name: 2-(3,5-dichlorophenyl)-1,3-benzoxazole-6-carboxylic acid

ChEBI definition: A member of the class of 1,3-benzoxazoles that is 1,3-benzoxazole-6-carboxylic acid in which the hydrogen at position 2 is replaced by a 3,5-dichlorophenyl group. Used (as its meglumine salt) for the amelioration of transthyretin-related hereditary amyloidosis.

Pharmacological roles (ChEBI): central nervous system drug.

Also known as: FX-1005, FX-1006, FX1006, Tafamidis, Vyndamax, TAFAMIDIS, Tafamidis; Vyndaqel, tafamidis

Parent form; salt/anhydrous children: CHEMBL2105675

Patent coverage: 266 distinct patent families (672 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 440 (65%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
TTR	transthyretin	Binding	8.7	0%	P02766

Broader ChEMBL bioactivity targets: 8 (assay-derived). Sample: Amine oxidase [flavin-containing] A, D(1A) dopamine receptor, Cannabinoid receptor 1, Delta-type opioid receptor, cGMP-inhibited 3’,5’-cyclic phosphodiesterase 3A, 3’,5’-cyclic-AMP phosphodiesterase 4D, Transthyretin, Albumin.

Bioactivity

ChEMBL activities: 33 potent at pChembl ≥ 5 of 40 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
TTR	8.57	EC50	2.7	nM	CHEMBL_ACT_25500498
TTR	8.57	EC50	2.7	nM	CHEMBL_ACT_25500499
TTR	8.57	EC50	2.7	nM	CHEMBL_ACT_25500500
TTR	8.52	Kd	3	nM	CHEMBL_ACT_22795686
TTR	8.36	Kd	4.4	nM	CHEMBL_ACT_18557074
TTR	8.03	Kd	9.3	nM	CHEMBL_ACT_16761942
TTR	8	Kd	9.9	nM	CHEMBL_ACT_24670670
TTR	7.44	Kd	36	nM	CHEMBL_ACT_29145685
TTR	6.86	Kd	138	nM	CHEMBL_ACT_25987488
TTR	6.7	Kd	200	nM	CHEMBL_ACT_23263056
TTR	6.58	Kd	260	nM	CHEMBL_ACT_24670720
TTR	6.58	Kd	260	nM	CHEMBL_ACT_25666926
TTR	6.56	Kd	274	nM	CHEMBL_ACT_16761939
TTR	6.56	Kd	278	nM	CHEMBL_ACT_18091476
TTR	6.56	Kd	278	nM	CHEMBL_ACT_22795687
TTR	6.43	Kd	370	nM	CHEMBL_ACT_18091489
TTR	6.39	IC50	410	nM	CHEMBL_ACT_24355276
TTR	5.98	Kd	1050	nM	CHEMBL_ACT_19263640
TTR	5.81	IC50	1550	nM	CHEMBL_ACT_16815338
TTR	5.79	IC50	1620	nM	CHEMBL_ACT_16815332
TTR	5.66	Ki	2200	nM	CHEMBL_ACT_29145614
ALB	5.64	Kd	2300	nM	CHEMBL_ACT_18557082
ALB	5.6	Kd	2500	nM	CHEMBL_ACT_18557083
TTR	5.58	IC50	2600	nM	CHEMBL_ACT_23263028
TTR	5.55	Kd	2800	nM	CHEMBL_ACT_19263637
TTR	5.51	IC50	3100	nM	CHEMBL_ACT_16761949
TTR	5.51	IC50	3090	nM	CHEMBL_ACT_16761967
TTR	5.51	Ki	3100	nM	CHEMBL_ACT_25666884
TTR	5.51	IC50	3100	nM	CHEMBL_ACT_27718133
TTR	5.46	IC50	3490	nM	CHEMBL_ACT_25690707

Target pathways

Aggregated over 1 target gene(s): TTR.

Top Reactome pathways

6 total, by targets touching each:

Pathway	Targets	Genes
The canonical retinoid cycle in rods (twilight vision)	1	TTR
Non-integrin membrane-ECM interactions	1	TTR
Neutrophil degranulation	1	TTR
Retinoid metabolism and transport	1	TTR
Amyloid fiber formation	1	TTR
Defective visual phototransduction due to STRA6 loss of function	1	TTR

Dominant GO biological processes

GO term	Targets
retinoid metabolic process	1
negative regulation of glomerular filtration	1
purine nucleobase metabolic process	1
phototransduction, visible light	1
signal transduction	1

Indications & clinical

Indications

4 indications (3 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
cardiomyopathy	4	MONDO:0004994	EFO:0000318
amyloidosis	4	MONDO:0019065	EFO:1001875
hereditary amyloidosis	4	MONDO:0018634	MONDO:0019438
familial amyloid neuropathy	3	MONDO:0007100	EFO:0004129

Clinical trials

Total trials: 27.

Phase distribution

Phase	Trials
PHASE1	14
PHASE3	5
Not specified	5
PHASE4	3

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05489523	PHASE4	RECRUITING	Safety, Efficacy, and Pharmacokinetics of Tafamidis in Patients With Transthyretin-mediated Amyloidosis Post Orthotopic Heart Transplantation
NCT04814186	PHASE4	COMPLETED	A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
NCT04963985	PHASE4	UNKNOWN	The Effect of Tafamidis on Transthyretin Stabilization, Safety, Tolerability and Efficacy in Transthyretin Amyloid Polyneuropathy Patients
NCT00925002	PHASE3	COMPLETED	Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis
NCT00935012	PHASE3	COMPLETED	Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy
NCT01435655	PHASE3	COMPLETED	The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin
NCT01994889	PHASE3	COMPLETED	Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy
NCT02791230	PHASE3	COMPLETED	Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy
NCT07470866	PHASE1	RECRUITING	A Study to Compare the Amount of Two Forms of Tafamidis in the Blood of Healthy Adults Under Fed Conditions
NCT07587697	PHASE1	RECRUITING	A Study to Compare the Amount of Two Forms of Tafamidis in the Blood of Healthy Adults Under Fasting Conditions
NCT01369836	PHASE1	COMPLETED	Study Of Single Doses Of PF-06291826 (Tafamidis) In Japanese And Western Subjects
NCT01655511	PHASE1	COMPLETED	Safety And Pharmacokinetic Assessment Of Orally Administered Tafamidis In Healthy Volunteers
NCT02189330	PHASE1	COMPLETED	A Study To Estimate How Much Tafamidis Created In Different Ways Can Be Measured in Blood Samples
NCT02217813	PHASE1	COMPLETED	A Bioequivalence Study Comparing Two Different Tafamidis Formulations
NCT02406560	PHASE1	COMPLETED	A Study Comparing The Amounts of Tafamidis In The Blood With Or Without Food
NCT02534740	PHASE1	COMPLETED	A Study Comparing Amounts of Tafamidis In The Blood With Or Without Food
NCT02697864	PHASE1	COMPLETED	A Study Comparing Amounts of Tafamidis In The Blood Without Food
NCT02746926	PHASE1	COMPLETED	A Study Comparing Amounts of Tafamidis (PF-06291826) in the Blood Without Food in Healthy Volunteers
NCT03266705	PHASE1	COMPLETED	A Study Comparing Amounts of 2 Different Forms of Tafamidis (PF-6291826) in the Blood
NCT03280173	PHASE1	COMPLETED	A Study Comparing the Amounts of 2 Different Forms of Tafamidis (PF06291826), Both With and Without Food, In the Blood
NCT03662191	PHASE1	COMPLETED	Phase I Safety, Pharmacokinetics and Bioavailability Study Comparing Rate and Extent of Absorption of Two Different Forms of Tafamidis (PF-6291826)
NCT04253353	PHASE1	COMPLETED	A Drug-Drug Interaction Study To Estimate The Effect Of Tafamidis On Rosuvastatin Pharmacokinetics
NCT05139680	Not specified	COMPLETED	A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis
NCT05560555	Not specified	COMPLETED	Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045.
NCT06086353	Not specified	COMPLETED	A Study to Learn About Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in India
NCT06321523	Not specified	COMPLETED	A Study to Learn About the Study Medicine Called Tafamidis 61mg in People Diagnosed With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
NCT06393465	Not specified	COMPLETED	Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

37 molecules share ≥1 primary target. Top 37 by shared-target count:

Molecule	Source	Status	Shared targets
ACORAMIDIS	ChEMBL	Phase 4 (approved)	TTR
AMLEXANOX	ChEMBL	Phase 4 (approved)	TTR
BENZBROMARONE	ChEMBL	Phase 4 (approved)	TTR
BENZIODARONE	ChEMBL	Phase 4 (approved)	TTR
BITHIONOL	ChEMBL	Phase 4 (approved)	TTR
DASATINIB	ChEMBL	Phase 4 (approved)	TTR
DEXTROTHYROXINE	ChEMBL	Phase 4 (approved)	TTR
DICLOFENAC	ChEMBL	Phase 4 (approved)	TTR
DIFLUNISAL	ChEMBL	Phase 4 (approved)	TTR
GEMFIBROZIL	ChEMBL	Phase 4 (approved)	TTR
LEVOTHYROXINE	ChEMBL	Phase 4 (approved)	TTR
MECLOFENAMIC ACID	ChEMBL	Phase 4 (approved)	TTR
TOLCAPONE	ChEMBL	Phase 4 (approved)	TTR
TRICLABENDAZOLE	ChEMBL	Phase 4 (approved)	TTR
TRICLOSAN	ChEMBL	Phase 4 (approved)	TTR
CAFFEIC ACID	ChEMBL	Phase 3	TTR
DIACEREIN	ChEMBL	Phase 3	TTR
EPIGALOCATECHIN GALLATE	ChEMBL	Phase 3	TTR
RESVERATROL	ChEMBL	Phase 3	TTR
ACECLOFENAC	ChEMBL	Phase 2	TTR
DAIDZEIN	ChEMBL	Phase 2	TTR
FLUFENAMIC ACID	ChEMBL	Phase 2	TTR
GENISTEIN	ChEMBL	Phase 2	TTR
LUTEOLIN	ChEMBL	Phase 2	TTR
NIFLUMIC ACID	ChEMBL	Phase 2	TTR
PTEROSTILBENE	ChEMBL	Phase 2	TTR
TOLFENAMIC ACID	ChEMBL	Phase 2	TTR
XANTHOHUMOL	ChEMBL	Phase 2	TTR
albuterol	PubChem	Approved	TTR
Amiodarone	PubChem	Approved	TTR
Bromhexine	PubChem	Approved	TTR
Capsaicin	PubChem	Approved	TTR
Carbinoxamine	PubChem	Approved	TTR
Chlorogenic Acid	PubChem	Approved	TTR
Crizotinib	PubChem	Approved	TTR
Nitisinone	PubChem	Approved	TTR
Tafamidis Meglumine	PubChem	Approved	TTR