U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Details

Stereochemistry ABSOLUTE
Molecular Formula C61H63N9O17S4.4H
Molecular Weight 1326.495
Optical Activity UNSPECIFIED
Defined Stereocenters 1 / 1
E/Z Centers 3
Charge 0

SHOW SMILES / InChI
Structure of PAFOLACIANINE

SMILES

[H+].[H+].[H+].[H+].CC1(C)\C(=C/C=C2\CCCC(\C=C\C3=[N+](CCCCS([O-])(=O)=O)C4=CC=C(C=C4C3(C)C)S([O-])(=O)=O)=C2OC5=CC=C(C[C@H](NC(=O)C6=CC=C(NCC7=NC8=C(N=C7)N=C(N)NC8=O)C=C6)C([O-])=O)C=C5)N(CCCCS([O-])(=O)=O)C9=CC=C(C=C19)S([O-])(=O)=O

InChI

InChIKey=PDXNSXLPXJFETD-DYVQZXGMSA-N
InChI=1S/C61H67N9O17S4/c1-60(2)46-33-44(90(81,82)83)22-24-49(46)69(28-5-7-30-88(75,76)77)51(60)26-16-38-10-9-11-39(17-27-52-61(3,4)47-34-45(91(84,85)86)23-25-50(47)70(52)29-6-8-31-89(78,79)80)54(38)87-43-20-12-37(13-21-43)32-48(58(73)74)66-56(71)40-14-18-41(19-15-40)63-35-42-36-64-55-53(65-42)57(72)68-59(62)67-55/h12-27,33-34,36,48H,5-11,28-32,35H2,1-4H3,(H9-,62,63,64,66,67,68,71,72,73,74,75,76,77,78,79,80,81,82,83,84,85,86)/t48-/m0/s1

HIDE SMILES / InChI

Molecular Formula C61H67N9O17S4
Molecular Weight 1326.495
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry
Defined Stereocenters 1 / 1
E/Z Centers 3
Optical Activity UNSPECIFIED

OTL-38 (OTL-0038, Pafolacianine), a fluorescent-labelled folate receptor-α (FRα) targeted imaging agent that accumulates in vivo in tumor cells expressing FR. In 2014, the OTL-38 molecule was granted orphan drug status which can be given to the maker of a drug that treats rare conditions or diseases and offers protection from competition for a period of time. OTL-38 under the brand name Cytalux was approved by the U.S. Food and Drug Administration (FDA) on 29 November 2021, as an additional approach that can be used to identify malignant lesions and to ensure the total resection of the tumors in ovarian cancer patients. Cytalux is a fluorescent drug that targets folate receptor which may be overexpressed in ovarian cancer. Pafolacianine binds to FR-expressing cancer cells with ~1 nM affinity, internalizes via receptor mediated endocytosis, and concentrates in FR-positive cancer tissues. Pafolacianine absorbs light in the near-infrared region within a range of 760 nm to 785 nm with peak absorption of 776 nm and emits fluorescence within a range of 790 nm to 815 nm with a peak emission of 796 nm.

Approval Year

Targets

Targets

Primary TargetPharmacologyConditionPotency
Target ID: P15328
Gene ID: 2348.0
Gene Symbol: FOLR1
Target Organism: Homo sapiens (Human)
0.8 null [Other]
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Diagnostic
CYTALUX

Approved Use

CYTALUX is an optical imaging agent indicated in adult patients with ovarian cancer as an adjunct for intraoperative identification of malignant lesions.

Launch Date

2021
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
59.1 ng/mL
3.2 mg single, intravenous
dose: 3.2 mg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: UNKNOWN
age: UNKNOWN
sex: UNKNOWN
food status: UNKNOWN
61.7 ng/mL
0.025 mg/kg single, intravenous
dose: 0.025 mg/kg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
173 ng/mL
0.05 mg/kg single, intravenous
dose: 0.05 mg/kg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
458 ng/mL
0.1 mg/kg single, intravenous
dose: 0.1 mg/kg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
606 ng/mL
0.2 mg/kg single, intravenous
dose: 0.2 mg/kg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
63.6 ng × h/mL
3.2 mg single, intravenous
dose: 3.2 mg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: UNKNOWN
age: UNKNOWN
sex: UNKNOWN
food status: UNKNOWN
59.2 ng × h/mL
0.025 mg/kg single, intravenous
dose: 0.025 mg/kg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
250 ng × h/mL
0.05 mg/kg single, intravenous
dose: 0.05 mg/kg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
830 ng × h/mL
0.1 mg/kg single, intravenous
dose: 0.1 mg/kg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
1250 ng × h/mL
0.2 mg/kg single, intravenous
dose: 0.2 mg/kg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
0.44 h
3.2 mg single, intravenous
dose: 3.2 mg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: UNKNOWN
age: UNKNOWN
sex: UNKNOWN
food status: UNKNOWN
2.6 h
0.2 mg/kg single, intravenous
dose: 0.2 mg/kg
route of administration: Intravenous
experiment type: SINGLE
co-administered:
OTL-38 plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
Doses

Doses

DosePopulationAdverse events​
0.2 mg/kg single, intravenous
Highest studied dose
Dose: 0.2 mg/kg
Route: intravenous
Route: single
Dose: 0.2 mg/kg
Sources:
healthy, ADULT
Health Status: healthy
Age Group: ADULT
Sex: unknown
Food Status: UNKNOWN
Sources:
0.025 mg/kg single, intravenous
Recommended
Dose: 0.025 mg/kg
Route: intravenous
Route: single
Dose: 0.025 mg/kg
Sources:
unhealthy, ADULT
Health Status: unhealthy
Age Group: ADULT
Sex: F
Food Status: UNKNOWN
Sources:
Disc. AE: Vomiting, Nausea...
AEs leading to
discontinuation/dose reduction:
Vomiting (2 pt)
Nausea (3 patients)
Hypersensitivity (2 patients)
Rash (1 pt)
Sources:
0.025 mg/kg single, intravenous
Recommended
Dose: 0.025 mg/kg
Route: intravenous
Route: single
Dose: 0.025 mg/kg
Sources:
unhealthy, ADULT
Health Status: unhealthy
Age Group: ADULT
Sex: M+F
Food Status: UNKNOWN
Sources:
Disc. AE: AE, AE...
AEs leading to
discontinuation/dose reduction:
AE (13 patients)
AE (41 patient)
Vomiting (<= 2, 2 patients)
Sources:
AEs

AEs

AESignificanceDosePopulation
Rash 1 pt
Disc. AE
0.025 mg/kg single, intravenous
Recommended
Dose: 0.025 mg/kg
Route: intravenous
Route: single
Dose: 0.025 mg/kg
Sources:
unhealthy, ADULT
Health Status: unhealthy
Age Group: ADULT
Sex: F
Food Status: UNKNOWN
Sources:
Hypersensitivity 2 patients
Disc. AE
0.025 mg/kg single, intravenous
Recommended
Dose: 0.025 mg/kg
Route: intravenous
Route: single
Dose: 0.025 mg/kg
Sources:
unhealthy, ADULT
Health Status: unhealthy
Age Group: ADULT
Sex: F
Food Status: UNKNOWN
Sources:
Vomiting 2 pt
Disc. AE
0.025 mg/kg single, intravenous
Recommended
Dose: 0.025 mg/kg
Route: intravenous
Route: single
Dose: 0.025 mg/kg
Sources:
unhealthy, ADULT
Health Status: unhealthy
Age Group: ADULT
Sex: F
Food Status: UNKNOWN
Sources:
Nausea 3 patients
Disc. AE
0.025 mg/kg single, intravenous
Recommended
Dose: 0.025 mg/kg
Route: intravenous
Route: single
Dose: 0.025 mg/kg
Sources:
unhealthy, ADULT
Health Status: unhealthy
Age Group: ADULT
Sex: F
Food Status: UNKNOWN
Sources:
AE 13 patients
Disc. AE
0.025 mg/kg single, intravenous
Recommended
Dose: 0.025 mg/kg
Route: intravenous
Route: single
Dose: 0.025 mg/kg
Sources:
unhealthy, ADULT
Health Status: unhealthy
Age Group: ADULT
Sex: M+F
Food Status: UNKNOWN
Sources:
AE 41 patient
Disc. AE
0.025 mg/kg single, intravenous
Recommended
Dose: 0.025 mg/kg
Route: intravenous
Route: single
Dose: 0.025 mg/kg
Sources:
unhealthy, ADULT
Health Status: unhealthy
Age Group: ADULT
Sex: M+F
Food Status: UNKNOWN
Sources:
Vomiting <= 2, 2 patients
Disc. AE
0.025 mg/kg single, intravenous
Recommended
Dose: 0.025 mg/kg
Route: intravenous
Route: single
Dose: 0.025 mg/kg
Sources:
unhealthy, ADULT
Health Status: unhealthy
Age Group: ADULT
Sex: M+F
Food Status: UNKNOWN
Sources:
Overview

Overview

Drug as perpetrator​

Drug as perpetrator​

TargetModalityActivityMetaboliteClinical evidence
likely
likely
likely
likely
likely
likely
likely
likely
likely
likely
likely
likely
likely
likely
likely
likely
likely
likely
no
Drug as victim

Drug as victim

TargetModalityActivityMetaboliteClinical evidence
inconclusive
inconclusive
no
no
no
no
no
no
yes
yes
yes
Tox targets

Tox targets

PubMed

PubMed

TitleDatePubMed
Intraoperative Tumor Detection Using Pafolacianine.
2022-10-25
Folate receptor-α targeted near-infrared fluorescence imaging in high-risk endometrial cancer patients: a tissue microarray and clinical feasibility study.
2018-01-02
Synthesis and composition of amino acid linking groups conjugated to compounds used for the targeted imaging of tumors: a patent evaluation of US20160011199A1.
2016-11
Patents

Sample Use Guides

Recommended dosage of CYTALUX is 0.025 mg/kg administered intravenously over 60 minutes 1 hour to 9 hours prior to surgery.
Route of Administration: Intravenous
Pafolacianine binds to FR-expressing cancer cells with ~1 nM affinity.
Substance Class Chemical
Created
by admin
on Mon Mar 31 18:50:10 GMT 2025
Edited
by admin
on Mon Mar 31 18:50:10 GMT 2025
Record UNII
F7BD3Z4X8L
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
PAFOLACIANINE
USAN   INN  
Official Name English
OTL-0038
Preferred Name English
OTL-38
Common Name English
PAFOLACIANINE [USAN]
Common Name English
Pafolacianine [WHO-DD]
Common Name English
2-(2-(2-(4-((2S)-2-(4-(((2-AMINO-4-OXO-3,4-DIHYDROPTERIDIN-6-YL)METHYL)AMINO)BENZAMIDO)-2-CARBOXYETHYL)PHENOXY)-3-(2-(3,3-DIMETHYL-5-SULFO-1-(4-SULFOBUTYL)-1,3-DIHYDRO-2H-INDOL-2-YLIDENE)ETHYLIDENE)CYCLOHEX-1-EN-1-YL)ETHENYL)-3,3-DIMETHYL-5-SULFO-1-(4-SU
Systematic Name English
OTL0038
Code English
pafolacianine [INN]
Common Name English
3H-INDOLIUM, 2-(2-(2-(4-((2S)-2-((4-(((2-AMINO-3,4-DIHYDRO-4-OXO-6-PTERIDINYL)METHYL)AMINO)BENZOYL)AMINO)-2-CARBOXYETHYL)PHENOXY)-3-(2-(1,3-DIHYDRO-3,3-DIMETHYL-5-SULFO-1-(4-SULFOBUTYL)-2H-INDOL-2-YLIDENE)ETHYLIDENE)-1-CYCLOHEXEN-1-YL)ETHENYL)-3,3-DIMETH
Systematic Name English
OTL38
Code English
Classification Tree Code System Code
FDA ORPHAN DRUG 458714
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
Code System Code Type Description
INN
11569
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
DAILYMED
F7BD3Z4X8L
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
RXCUI
2586857
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
DRUG BANK
DB15413
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
USAN
HI-69
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
NCI_THESAURUS
C175834
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
CAS
1628423-76-6
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
FDA UNII
F7BD3Z4X8L
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
SMS_ID
300000013880
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
PUBCHEM
135565623
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
WIKIPEDIA
Pafolacianine
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
PRIMARY
CAS
1983160-11-7
Created by admin on Mon Mar 31 18:50:10 GMT 2025 , Edited by admin on Mon Mar 31 18:50:10 GMT 2025
NO STRUCTURE GIVEN
Related Record Type Details
SALT/SOLVATE -> PARENT
TARGET->LIGAND
Imaging agent
Related Record Type Details
ACTIVE MOIETY