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Lirafugratinib

Previously treated FGFR2 fusion or rearrangement-positive cholangiocarcinoma

Also known as Lyrfigtu, RLY-4008
Regulatory submission
NDA submitted January 2026
158 sources

Section 1 of 6

Terms and abbreviations

1 evidence topic · 0 sources

AbbreviationTerm
14CCarbon-14
1LFirst line
1LOTFirst line of therapy
2LSecond line
2LOTSecond line of therapy
3LThird line
3LOTThird line of therapy
5-FUFluorouracil
5-FU/LVFluorouracil and leucovorin
5FUFluorouracil
ABCAdvanced biliary cancer
ABC-06Advanced Biliary Cancer 06 trial
ABCB1ATP-binding cassette subfamily B member 1 (P-glycoprotein)
ABCG2ATP-binding cassette subfamily G member 2 (breast cancer resistance protein)
ADCAntibody-drug conjugate
AEAdverse event
AHCYL1Adenosylhomocysteinase like 1
AHFSAmerican Hospital Formulary Service
aHRAdjusted hazard ratio
AJCCAmerican Joint Committee on Cancer
AKTProtein kinase B
ALKAnaplastic lymphoma kinase
ANOVAAnalysis of variance
ARID1AT-rich interaction domain 1
ARID1AAT-rich interaction domain 1A
ASCActive symptom control
ASCOAmerican Society of Clinical Oncology
ATCAnatomical Therapeutic Chemical classification
ATMAtaxia telangiectasia mutated
ATPAdenosine triphosphate
AUCArea under the concentration-time curve
AUC0-infArea under the concentration-time curve from time zero to infinity
B+TBGJ398 plus trametinib
BAIAP2L1BAR/IMD domain containing adaptor protein 2 like 1
BAP1BRCA1-associated protein 1
BCRPBreast cancer resistance protein
BGJ398Development code for infigratinib
BICC1BicC family RNA binding protein 1
BICRBlinded independent central review
BIDTwice daily
BMIBody mass index
BRAFB-Raf proto-oncogene, serine/threonine kinase
BSEPBile salt export pump
BTCBiliary tract cancer
BTTBiliary tract tumor
C13D1Cycle 13, day 1
C3D1Cycle 3, day 1
CA 19-9Carbohydrate antigen 19-9
CA19-9Carbohydrate antigen 19-9
CADTHCanadian Agency for Drugs and Technologies in Health
CAFsCancer-associated fibroblasts
CAPCollege of American Pathologists
CCACholangiocarcinoma
CCDC6Coiled-coil domain containing 6
CDA-AMCCanada's Drug Agency
CDKN2ACyclin-dependent kinase inhibitor 2A
CDKN2BCyclin-dependent kinase inhibitor 2B
CDxCompanion diagnostic
CEACarcinoembryonic antigen
cfDNACell-free DNA
CFRCode of Federal Regulations
CGPComprehensive genomic profiling
ChTChemotherapy
CIConfidence interval
CLcrCreatinine clearance
CLIAClinical Laboratory Improvement Amendments
CmaxMaximum plasma concentration
CNSCentral nervous system
CRComplete response
CTComputed tomography
CTCAECommon Terminology Criteria for Adverse Events
ctDNACirculating tumor DNA
CTNNB1Catenin beta 1
CUPCancer of unknown primary
CYPCytochrome P450
CYP1A2Cytochrome P450 1A2
CYP2B6Cytochrome P450 2B6
CYP2C19Cytochrome P450 2C19
CYP2C8Cytochrome P450 2C8
CYP2C9Cytochrome P450 2C9
CYP2D6Cytochrome P450 2D6
CYP2J2Cytochrome P450 2J2
CYP3ACytochrome P450 3A
CYP3A4Cytochrome P450 3A4
Cys491Cysteine 491
d/pCCADistal or perihilar cholangiocarcinoma
dCCADistal cholangiocarcinoma
DCRDisease control rate
Debio1347Development code for zoligratinib
DFSDisease-free survival
dMMRMismatch repair deficient
DNADeoxyribonucleic acid
DORDuration of response
DoRDuration of response
EAGExternal assessment group
eAPCEstimated annual percent change
ECCExtrahepatic cholangiocarcinoma
eCCAExtrahepatic cholangiocarcinoma
ECGElectrocardiogram
ECMExtracellular matrix
ECOGEastern Cooperative Oncology Group
EFSEvent-free survival
EGFEpidermal growth factor
EGFREpidermal growth factor receptor
ELE-4008-202Protocol identifier of the ReFocus202 study
eLTSExtended long-term survivors
EMAEuropean Medicines Agency
EMPExtended molecular profiling
EORTCEuropean Organisation for Research and Treatment of Cancer
ePosterElectronic poster
EQ-5DEuroQol 5 Dimension
EQ-5D-5LEuroQol 5 Dimension, 5 level version
ERBBErythroblastic oncogene B
ERBB2Erb-b2 receptor tyrosine kinase 2
ERCEndoscopic retrograde cholangiography
ERCPEndoscopic retrograde cholangiopancreatography
ERKExtracellular signal-regulated kinase
ESCATESMO Scale for Clinical Actionability of Molecular Targets
ESMOEuropean Society for Medical Oncology
ESMO-MCBSEuropean Society for Medical Oncology Magnitude of Clinical Benefit Scale
ETEastern Time
EUEuropean Union
EUSEndoscopic ultrasonography
evNGSExternally validated next-generation sequencing
F-TKIFGFR-specific tyrosine kinase inhibitor
f/rFusion or rearrangement
F1CDxFoundationOne CDx
FDAUS Food and Drug Administration
FDG-PETFluorodeoxyglucose positron emission tomography
FFFGFR2 fusion
FGFFibroblast growth factor
FGFRFibroblast growth factor receptor
FGFR1Fibroblast growth factor receptor 1
FGFR2Fibroblast growth factor receptor 2
FGFR3Fibroblast growth factor receptor 3
FGFR4Fibroblast growth factor receptor 4
FGFRiFibroblast growth factor receptor inhibitor
FIGHT-202Phase 2 trial of pemigatinib in previously treated cholangiocarcinoma
FIHFirst-in-human
FISHFluorescence in situ hybridization
FNAFine-needle aspiration
FOENIX-CCA2Phase 2 trial of futibatinib in FGFR2 fusion or rearrangement-positive intrahepatic cholangiocarcinoma
FOLFOXFolinic acid, fluorouracil, and oxaliplatin
FRFold-reversal
FUDRFloxuridine
GAGenetic aberration
GBCGallbladder cancer
GemCisGemcitabine and cisplatin
GHSGlobal health status
HAIPHepatic artery infusion pump
HBVHepatitis B virus
HCCHepatocellular carcinoma
HCRUHealthcare resource utilization
HCVHepatitis C virus
HEK293Human embryonic kidney 293 cells
HER2Human epidermal growth factor receptor 2
HLBHLB Co., Ltd., majority owner of Elevar Therapeutics
HRHazard ratio; hormone receptor where written HR+
IC50Half-maximal inhibitory concentration
ICCIntrahepatic cholangiocarcinoma
iCCAIntrahepatic cholangiocarcinoma
ICERIncremental cost-effectiveness ratio
IDH1Isocitrate dehydrogenase 1
IDH2Isocitrate dehydrogenase 2
IHCImmunohistochemistry
IHCCIntrahepatic cholangiocarcinoma
IL-1βInterleukin 1 beta
INDInvestigational new drug application
INHBIncremental net health benefit
INMBIncremental net monetary benefit
IPNBIntraductal papillary neoplasm of the bile duct
IQRInterquartile range
IRCIndependent review committee
IRRIncidence rate ratio
IVIntravenous
KEYNOTE-966Phase 3 trial of pembrolizumab plus gemcitabine and cisplatin in advanced biliary tract cancer
KMKaplan-Meier
KRASKirsten rat sarcoma viral oncogene homolog
LBxLiquid biopsy
LDPLimited-duration prevalence
LLCLimited liability company
LoELevel of evidence
LOTLine of therapy
LSMLeast squares mean
LVLeucovorin
LYLife-year
MAICMatching-adjusted indirect comparison
MAPKMitogen-activated protein kinase
MATE1Multidrug and toxin extrusion protein 1
MATE2-KMultidrug and toxin extrusion protein 2-K
MBqMegabecquerel
MbxMetastatic-site biopsy
MCBSMagnitude of Clinical Benefit Scale
MDTBMultidisciplinary tumor board
MedDRAMedical Dictionary for Regulatory Activities
MEKMitogen-activated protein kinase kinase
MEK5Mitogen-activated protein kinase kinase 5
mFOLFOXModified folinic acid, fluorouracil, and oxaliplatin
MKNK2MAPK interacting serine/threonine kinase 2
mosMonths
mOSMedian overall survival
MRCPMagnetic resonance cholangiopancreatography
MRIMagnetic resonance imaging
MSI-HMicrosatellite instability-high
MTDMaximum tolerated dose
mTORMechanistic target of rapamycin
MYCMYC proto-oncogene
N/ANot applicable
NAFLDNon-alcoholic fatty liver disease
nal-IRILiposomal irinotecan
NALIRICCRandomized phase 2 trial of liposomal irinotecan with fluorouracil and leucovorin in biliary tract cancer
NCNot calculable
NCCNNational Comprehensive Cancer Network
NDANew drug application
NDCNational Drug Code
NENot estimable
NGSNext-generation sequencing
NHINational Health Insurance (Taiwan)
NHIANational Health Insurance Administration (Taiwan)
NHIRDNational Health Insurance Research Database (Taiwan)
NHSNational Health Service
NICENational Institute for Health and Care Excellence
NIFTYRandomized phase 2b trial of liposomal irinotecan with fluorouracil and leucovorin in biliary tract cancer
NOSNot otherwise specified
NRNot reached
NT$New Taiwan dollar
NTRKNeurotrophic tyrosine receptor kinase
OAT1Organic anion transporter 1
OAT3Organic anion transporter 3
OATP1B1Organic anion transporting polypeptide 1B1
OATP1B3Organic anion transporting polypeptide 1B3
OCTOptical coherence tomography
OCT1Organic cation transporter 1
OCT2Organic cation transporter 2
OROdds ratio
ORRObjective response rate
OSOverall survival
P-gpP-glycoprotein
pan-FGFRiInhibitor of all fibroblast growth factor receptor isoforms
PBRM1Polybromo 1
PbxPrimary tumor biopsy
pCCAPerihilar cholangiocarcinoma
pCODRpan-Canadian Oncology Drug Review
PDProgressive disease
PD-L1Programmed death-ligand 1
PDGFPlatelet-derived growth factor
PDyPharmacodynamics
pERCpCODR Expert Review Committee
pERKPhosphorylated extracellular signal-regulated kinase
PFProgression-free
pFGFRPhosphorylated fibroblast growth factor receptor
pFGFR2Phosphorylated fibroblast growth factor receptor 2
PFSProgression-free survival
PhAPheophorbide A
PI3KPhosphoinositide 3-kinase
PKPharmacokinetics
PPEPalmar-plantar erythrodysesthesia
PPPMPer patient per month
PRPartial response
PRKCAProtein kinase C alpha
PRKCBProtein kinase C beta
PROPatient-reported outcome
PROOF 301Phase 3 confirmatory trial of infigratinib in first-line FGFR2 fusion-positive cholangiocarcinoma
PSPerformance status
PSAProbabilistic sensitivity analysis
PSMPartitioned survival model
PTCPercutaneous transhepatic cholangiography
PTENPhosphatase and tensin homolog
ptsPatients
Q4Fourth quarter of a calendar year
QALYQuality-adjusted life-year
QDOnce daily
QDCOnce daily, continuous
QDDOnce daily, discontinuous
QLQ-BIL21EORTC Quality of Life Questionnaire, Cholangiocarcinoma and Gallbladder Cancer Module, 21 items
QLQ-C30EORTC Quality of Life Questionnaire Core 30
QoLQuality of life
QTcQT interval corrected for heart rate
QTcFQT interval corrected for heart rate by Fridericia's formula
RAFRapidly accelerated fibrosarcoma kinase
RASRat sarcoma virus GTPase
RECISTResponse Evaluation Criteria in Solid Tumors
RETRet proto-oncogene
RFSRelapse-free survival
RLY-4008Development code for lirafugratinib used by Relay Therapeutics
RLY-4008-101Protocol number of the ReFocus trial
RNARibonucleic acid
ROS1ROS proto-oncogene 1, receptor tyrosine kinase
RP2DRecommended phase 2 dose
RPEDRetinal pigment epithelial detachment
RT-PCRReverse transcription polymerase chain reaction
RTKReceptor tyrosine kinase
RTORReal-Time Oncology Review
rwPFSReal-world progression-free survival
S-1Tegafur, gimeracil, and oteracil
SATSubsequent anticancer therapy
SBRTStereotactic body radiotherapy
SDStable disease; standard deviation
SDF1Stromal cell-derived factor 1
SEERSurveillance, Epidemiology, and End Results
SMAD4SMAD family member 4
SN-387-Ethyl-10-hydroxycamptothecin, the active metabolite of irinotecan
SoCStandard of care
STAT3Signal transducer and activator of transcription 3
t1/2Half-life
TA1005NICE technology appraisal guidance 1005
TA1153NICE technology appraisal guidance 1153
TA722NICE technology appraisal guidance 722
TACC2Transforming acidic coiled-coil containing protein 2
TACC3Transforming acidic coiled-coil containing protein 3
TAPTumor area positivity
TAS-120Development code for futibatinib
TEAETreatment-emergent adverse event
TFTumor fraction
TKDTyrosine kinase domain
TKITyrosine kinase inhibitor
TmaxTime to maximum plasma concentration
TMETumor microenvironment
TNFTumor necrosis factor
TNMTumor, node, metastasis
TOPAZ-1Phase 3 trial of durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer
TP53Tumor protein p53
TRTotal radioactivity
TRAETreatment-related adverse event
TSGTumor suppressor gene
TTDTime to deterioration
TTPTime to progression
TWDNew Taiwan dollar
UKUnited Kingdom
USUnited States
US$United States dollar
USAUnited States of America
USDUnited States dollar
UVUltraviolet
VASVisual analogue scale
VATValue-added tax
VEGFVascular endothelial growth factor
VEGF-AVascular endothelial growth factor A
VEGF-CVascular endothelial growth factor C
WHOWorld Health Organization
wkWeek
wksWeeks
WNTWingless-related integration site
WTWild type
WTPWillingness to pay
ZIPZone Improvement Plan (United States postal code)