Expanded Indication for Pafolacianine Imaging Agent in Lung Cancer Approved by FDA


An expanded indication for pafolacianine as an imaging agent for patients with lung cancer has been approved by the FDA based on findings from the phase 3 ELUCIDATE trial.

The FDA has granted approval to an expanded indication for the targeted molecular imaging agent pafolacianine (Cytalux) for use in known or suspected lung cancer, according to a press release from On Target Laboratories.1

The expanded indication for pafolacianine is based on findings from the phase 3 ELUCIDATE trial (NCT04241315), which indicated that pafolacianine injections yielded efficacy and safety in patients scheduled to undergo thoracic surgery for lung cancer.

“During the ELUCIDATE trial, [pafolacianine] proved to be a valuable surgical tool with its ability to localize lung lesions that may have otherwise been missed,” Linda Martin, MD, MPH, chief of Thoracic Surgery at the University of Virginia School of Medicine, said in the press release. “[Pafolacianine] has potential to become standard of care in thoracic surgery because of the safety and efficacy demonstrated in the trial. It is an important tool for surgeons to consider for patients.”

Investigators of the single-dose, open-label phase 3 ELUCIDATE trial enrolled patients with lung cancer or lung neoplasms across 12 sites in the United States. Patients received 2 mg/mL of pafolacianine via injection with or without intraoperative imaging with a near infrared camera imaging system.

The primary end point of the ELUCIDATE trial was the rate of patients who had at least 1 clinically significant event following use of pafolacianine imaging.

Patients 18 years and older with a primary diagnosis or a high clinical suspicion of lung cancer were eligible to enroll on the study. Additional inclusion criteria included having a scheduled surgical thoracoscopy for diagnostic wedge resection followed by anatomic lung resection.

Patients who had any prior exposure to pafolacianine were unable to enroll on the study. Patients were also unsuitable for enrollment if they had any medical condition that could jeopardize their safety during treatment, a history of anaphylactic reactions to folate agents containing indocyanine green for near infrared imaging, a positive serum pregnancy test at screening, clinically significant abnormalities on electrocardiogram at screening, or impaired renal or liver function.

“Today’s approval of an expanded indication for [pafolacianine] marks an important step forward in the treatment landscape for lung cancer and for the more than 220,000 people in the U.S. who receive a lung cancer diagnosis each year,” Chris Barys, president and chief executive officer at On Target Laboratories, said. “We are proud to continue pioneering development of targeted intraoperative molecular imaging agents that illuminate cancers intraoperatively to enhance surgeons’ ability to see cancer in real time as they operate.”

Pafolacianine was previously approved by the FDA for detection of ovarian cancer during surgery in November 2021.2 The FDA based its approval on findings from a randomized trial, the data of which indicated that 26.9% of patients with ovarian cancer had at least 1 lesion detected with pafolacianine not observed through standard examinations.


  1. On Target Laboratories announces expanded indication of Cytalux® (pafolacianine) injection for detection of lung cancer during surgery. News release. On Target Laboratories. December 16, 2022. Accessed December 19, 2022. bit.ly/3HIHo49
  2. FDA approves new imaging drug to help identify ovarian cancer lesions. News release. FDA. November 29, 2021. Accessed December 19, 2022. bit.ly/3VauZZJ

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