Chemotherapy Regimen for Pancreatic Cancer Receives Approval for First-line Therapy 

The FDA has approved irinotecan liposome plus chemotherapy for the first-line treatment of metastatic pancreatic cancer. 

The U.S. Food and Drug Administration (FDA) has approved irinotecan liposome (Onivyde), in combination with the chemotherapeutics oxaliplatin, fluorouracil, and leucovorin (FOLFOX), for the first-line treatment of metastatic pancreatic cancer. 

Irinotecan inhibits an enzyme called topoisomerase that is important for DNA replication, thereby killing rapidly dividing cells, such as cancer cells. Irinotecan was first approved by the FDA in 1996 and is a mainstay of chemotherapy regimens for colorectal cancer.  

Illustration of a liposome. A spherical phospholipid bilayer is depicted by two concentric layers of blue phospholipid heads with brown phospholipid tails extending between them. The phospholipid bilayer surrounds a green aqueous sphere with circular red molecules floating inside. Irinotecan can packaged in liposomes to treat first-line metastatic pancreatic cancer.
Irinotecan, packaged in liposomes like the one shown here, is now approved to treat first-line metastatic pancreatic cancer. 

In 2015, a liposomal formulation of irinotecan—meaning irinotecan molecules are packaged in microscopic lipid bubbles for better delivery to certain tissues—was approved to treat pancreatic cancer that had advanced or relapsed following other treatments. The current approval allows patients to receive liposomal irinotecan as first-line treatment for metastatic pancreatic cancer. 

The approval was based on results from the randomized, multicenter, open-label, active-controlled, phase III NAPOLI 3 clinical trial. Researchers enrolled 770 patients with metastatic pancreatic cancer who had not yet received chemotherapy in the metastatic setting. Patients were randomly assigned (1:1) to receive irinotecan liposome plus FOLFOX or the chemotherapeutics gemcitabine and nab-paclitaxel. 

The median overall survival was 11.1 months for patients in the irinotecan liposome arm and 9.2 months for patients in the chemotherapy arm. The median progression-free survival was 7.4 months for patients in the irinotecan liposome arm and 5.6 months for patients in the chemotherapy arm. Both differences were statistically significant. 

The recommended dose of irinotecan liposome is 50 mg/m2 every two weeks, administered via a 90-minute intravenous infusion. Irinotecan liposome should be given before oxaliplatin, fluorouracil, and leucovorin. 

Pancreatic cancer is an aggressive tumor type with a generally poor prognosis and is expected to become the second-leading cause of cancer death in the United States by 2030. According to federal statistics, it was estimated that 64,050 individuals would be diagnosed with pancreatic cancer, and 50,550 patients would die of the disease in the United States in 2023. 

The FDA rendered its decision on February 13, 2024.