On November 29, 2021, the FDA accelerated the approval of pafolacianine (Cytalux; On Target Laboratories), a novel fluorescent imaging agent, for adults with ovarian cancer as an adjunct for identifying inoperable malignant lesions. Pafolacianine targets the folate receptor, which may be overexpressed in ovarian cancer. It is used with a near-infrared fluorescence imaging system approved specifically for use with pafolacianine. Pafolacianine received orphan drug designation for this indication.
The FDA approved pafolacianine based on a single-arm, multicenter, open-label study of 178 women with ovarian cancer or with high clinical suspicion of ovarian cancer who were scheduled to have surgical cytoreduction, interval debulking, or recurrent ovarian cancer surgery.
All the patients received pafolacianine. In addition to standard-of-care evaluation, 134 received fluorescence imaging evaluation; of these, 36 (26.9%) patients had at least 1 evaluable ovarian cancer lesion detected with pafolacianine that was not detected by standard evaluation. The false-positive rate with pafolacianine in detecting ovarian cancer lesions confirmed by central pathology was 20.2% (95% confidence interval, 13.7%-28.0%).
The most common (≥1%) adverse events were nausea, vomiting, abdominal pain, flushing, dyspepsia, chest discomfort, pruritus, and hypersensitivity.
The recommended pafolacianine dose is 0.025 mg/kg, administered intravenously over 60 minutes, 1 to 9 hours before surgery.