Pafolacianine

Mechanism-of-action classes: Fluorescence Contrast Activity; Imaging Contrast Activity.

• 1. INDICATIONS AND USAGE CYTALUX is indicated as an adjunct for intraoperative identification of: Malignant lesions in adult patients with ovarian cancer.ICD-10: C56.9

• None. None.contraindicated

For recommended testing, evaluations, and premedications, see Full Prescribing Information. ( 2.1 ) Recommended intravenous dosage of CYTALUX is: Adult Patients with Ovarian Cancer: 0.025 mg/kg over 60 minutes, 1 hour to 9 hours prior to surgery Adult Patients with Known or Suspected Cancer in the Lung: 0.025 mg/kg over 60 minutes, 1 hour to 24 hours prior to surgery. ( 2.2 ) For preparation, management of infusion-related reactions, and imaging information see Full Prescribing Information. CYTALUX should only be used by trained surgeons using FDA cleared imaging systems. ( 2.3 , 2.4 , 2.5 ) 2.1 Recommended Testing, Evaluations and Premedications Prior to Administration of CYTALUX Obtain a pregnancy test in females of reproductive potential and verify the absence of pregnancy prior to administration of CYTALUX [ see Warnings and Precautions (5.3) and Use in Specific Populations ( 8.1 , 8.3 ) ]. Discontinue folate, folic acid, or folate containing supplements 48 hours before administration of CYTALUX [ see Drug Interactions (7) ]. Consider administering antihistamines and/or anti-nausea medication for prophylaxis against infusion related reactions [ see Warnings and Precautions (5.1) ]. 2.2 Recommended Dosage and Administration Adult Patients with Ovarian Cancer The recommended dose of CYTALUX is a single intravenous infusion of 0.025 mg/kg diluted in 250 mL of 5% Dextrose Injection, administered over 60 minutes using a dedicated infusion line, 1 hour to 9 hours prior to surgery.

Infusion-Related Reactions: Interrupt the infusion and treat as necessary with antihistamines and/or nausea medications. ( 5.1 ) Risk of misinterpretation: Non-fluorescing tissue in the surgical field does not rule out the presence of tumor. Fluorescence may be seen in non-cancerous tissues. ( 5.2 ) Embryo-Fetal Toxicity: CYTALUX may cause fetal harm. Advise females of reproductive potential of the potential risk to a fetus. ( 5.3 , 8.1 , 8.3 ) Risk of Pafolacianine Aggregation and Infusion Reactions: Use only 5% Dextrose Injection for dilution. Do not use other diluents. ( 5.4 ) 5.1 Infusion-Related Reactions Adverse reactions including nausea, vomiting, abdominal pain, flushing, hypersensitivity, elevation in blood pressure, dyspepsia, and chest discomfort were reported in patients receiving CYTALUX in clinical studies. A total of 17% of patients experienced reactions during the administration of CYTALUX [see Adverse Reactions ( 6.1 )] . Reactions typically occurred within 15 minutes of the start of infusion. CYTALUX infusion interruption or discontinuation due to adverse reactions occurred in 11% of all patients. Treatment with antihistamines and/or anti-nausea medication may be used. If an adverse reaction occurs during administration, the infusion can be interrupted and resumed after treatment of the reaction [see Dosage and Administration ( 2.1 , 2.4 )] . 5.2 Risk of Misinterpretation Errors may occur with the use of CYTALUX during intraoperative fluorescence imaging to detect ovarian cancer and lesions in the lung, including false negatives and false positives.

The following clinically significant adverse reaction is described elsewhere in the labeling: • Infusion-Related Reactions [ see Warnings and Precautions ( 5.1 ) ] Most common adverse reactions (incidence ≥1%; ovarian and lung combined) included nausea, vomiting, abdominal pain, flushing, other infusion-related reactions, hypersensitivity, elevation in blood pressure, dyspepsia, and chest discomfort. ( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact On Target Laboratories at 1-844-434-9333 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of CYTALUX was evaluated in four open label clinical studies, two studies (N = 44 and N = 150) in patients with ovarian cancer and two studies (N = 100 and N = 112) in patients with known or suspected cancer in the lung. A total of 406 patients received 0.025 mg/kg of CYTALUX via intravenous administration. The demographic characteristics of the study population were 82% female (66% female in lung studies), mean age 64 years (range 26 to 89 years), 85% White, 6% Black or African American, 5% Asian, and 4% other race, 5% Hispanic or Latino, 92% Not Hispanic or Latino, and 3% unreported ethnicity. Adverse reactions that occurred in > 1% of patients are presented in Table 1. Table 1.

8.1 Pregnancy Risk Summary Based on its mechanism of action, pafolacianine may cause fetal harm when administered to a pregnant woman [see Clinical Pharmacology ( 12.1 )] . There are no available human data to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. No adverse developmental effects were observed in rats and rabbits with intravenous administration of pafolacianine during organogenesis (embryofetal development) at doses up to 158-fold (rat) and 570-fold (rabbit) the recommended human dose of 0.025 mg/kg based on AUC, otherwise 9.6 and 38.4-fold based on human equivalent dose (HED) ( see Data ). The estimated background risk of major birth defects and miscarriage for the indicated populations are unknown. All pregnancies have a background risk of birth defects, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

• Nov 29, 2021NDANDA214907On Target Labs

1. 1Nausea2551%
2. 2Vomiting1429%
3. 3Chest Discomfort48.2%
4. 4Drug Effect Less Than Expected48.2%
5. 5Dyspnoea48.2%
6. 6Pruritus48.2%
7. 7False Positive Investigation Result36.1%
8. 8Flushing36.1%
9. 9Wheezing36.1%
10. 10Chest Pain24.1%
11. 11Death24.1%
12. 12Hyperhidrosis24.1%
13. 13Muscle Spasms24.1%
14. 14Product Use In Unapproved Indication24.1%
15. 15Stridor24.1%

The 10 most recently updated of 17 ClinicalTrials.gov registrations naming Pafolacianine as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Safety and Feasibility of Intraoperative Visualization With Cytalux in Children
  Recruiting · Phase 1 · Interventional · 10 enrolled · Ann & Robert H Lurie Children's Hospital of Chicago
  NCT06235125updated 2026-06-05
• Single Dose Investigator Initiated Pilot Study to Investigate CYTALUX (Pafolacianine) for Intraoperative Detection of Malignant Tissue in Subjects Undergoing Surgical Resection for Cancer.
  Recruiting · Phase 2 · Interventional · 50 enrolled · John Waters
  NCT07039526updated 2026-06-05
• CYTALUX™for the Intraoperative Imaging of Prostate Cancer
  Completed · Phase 2 · Interventional · 9 enrolled · Indiana University
  NCT06434909updated 2026-06-01
• An Imaging Agent (Pafolacianine) for Identifying Lesions in Pediatric Patients With Primary or Metastatic Solid Tumors
  Recruiting · Phase 3 · Interventional · 20 enrolled · Mayo Clinic
  NCT06915727updated 2026-04-01
• CYTALUX Dose Extension Study
  Not yet recruiting · Phase 4 · Interventional · 106 enrolled · On Target Laboratories, LLC
  NCT07469202updated 2026-03-13
• PhI Pilot Study Pafolacianine Inject for Intraoperative Imaging on Outcomes of GI Cancer Peritoneal Carcinomatosis
  Recruiting · Early phase 1 · Interventional · 10 enrolled · University of California, Irvine
  NCT06511037updated 2026-02-27
• CYTALUX for Intraoperative Imaging of Patients With Endometrial Cancer
  Recruiting · Early phase 1 · Interventional · 10 enrolled · Abramson Cancer Center at Penn Medicine
  NCT07278986updated 2025-12-12
• Intraoperative Imaging of Gastrointestinal Malignancies Using Pafolacianine (CYTALUX™)
  Recruiting · Phase 4 · Interventional · 15 enrolled · Cedars-Sinai Medical Center
  NCT07124351updated 2025-09-15
• OTL38 Injection for Intraoperative Imaging of Folate Receptor Positive Lung Nodules
  Completed · Phase 2 · Interventional · 100 enrolled · On Target Laboratories, LLC
  NCT02872701updated 2023-03-24
• ELUCIDATE: Enabling Lung Cancer Identification Using Folate Receptor Targeting
  Completed · Phase 3 · Interventional · 112 enrolled · On Target Laboratories, LLC
  NCT04241315updated 2023-03-23

Frequently asked questions

How does Pafolacianine work?

Mechanism-of-action classes: Fluorescence Contrast Activity; Imaging Contrast Activity.

What is Pafolacianine used for?

According to FDA labeling, Pafolacianine carries indications including: 1. INDICATIONS AND USAGE CYTALUX is indicated as an adjunct for intraoperative identification of: Malignant lesions in adult patients with ovarian cancer.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Pafolacianine?

Pafolacianine is classified as Other diagnostic agents, Optical Imaging Agent, Fluorescence Contrast Activity, Imaging Contrast Activity.

What are the contraindications for Pafolacianine?

Pafolacianine labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.