FDA approval of liposomal irinotecan was based on the results of an open-label trial (NAPOLI-1) in 417 patients with metastatic pancreatic ductal adenocarcinoma whose disease progressed after gemcitabine-based therapy. Patients were randomized to receive either liposomal irinotecan alone, fluorouracil and leucovorin alone, or liposomal irinotecan in combination with fluorouracil and leucovorin. Median overall survival, the primary endpoint, was significantly longer with all three drugs (6.1 months), compared to fluorouracil and leucovorin alone (4.2 months) and liposomal irinotecan alone (4.9 months). The most frequent severe (grade 3 or 4) adverse effects of the liposomal irinotecan-containing regimen were neutropenia, diarrhea, vomiting, and fatigue.2 Life-threatening diarrhea has also occurred in patients receiving the 3-drug combination.

Onivyde is available in 43 mg/10 mL single-dose vials. The recommended dosage is 70 mg/m² administered intravenously over 90 minutes every 2 weeks; leucovorin and fluorouracil should be administered after liposomal irinotecan. The recommended starting dose of Onivyde for patients who are homozygous for the UGT1A1*28 allele is 50 mg/m²; the dose can be increased to 70 mg/m² as tolerated. The labeling specifies a number of dosage adjustments that should be made when adverse effects occur. One dose of Onivyde costs $4860.3