FDA Approves Liposomal Irinotecan for Advanced Pancreatic Cancer

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The U.S. Food and Drug Administration has approved irinotecan liposome injection (Onivyde), in combination with fluorouracil (5-FU) and leucovorin, to treat patients with metastatic pancreatic cancer who have been previously treated with gemcitabine-based chemotherapy.

The effectiveness of liposomal irinotecan was demonstrated in a three-arm, randomized, open-label study of 417 patients with metastatic pancreatic adenocarcinoma whose cancer had grown after receiving the chemotherapeutic drug gemcitabine or a gemcitabine-based therapy.1 The study was designed to determine whether patients receiving liposomal irinotecan plus 5-FU/leucovorin or liposomal irinotecan alone lived longer than those receiving 5-FU/leucovorin.

Patients treated with liposomal irinotecan plus 5-FU/leucovorin lived an average of 6.1 months, compared to 4.2 months for those treated with only 5-FU/leucovorin. There was no survival improvement for those who received only liposomal irinotecan compared to those who received 5-FU/leucovorin.

In addition, patients receiving liposomal irinotecan plus 5-FU/leucovorin had a delay in the amount of time to tumor growth compared to those who received 5-FU/leucovorin. The average time for those receiving liposomal irinotecan plus 5-FU/leucovorin was 3.1 months compared to 1.5 months for those receiving 5-FU/leucovorin.

Safety and Toxicity

The safety of liposomal irinotecan was evaluated in 398 patients who received either liposomal irinotecan with 5-FU/leucovorin, liposomal irinotecan alone, or 5-FU/leucovorin. The most common side effects of treatment with liposomal irinotecan included diarrhea, fatigue, vomiting, nausea, decreased appetite, stomatitis, and pyrexia. Liposomal irinotecan was also found to result in lymphopenia and neutropenia. Death due to sepsis following neutropenia has been reported in patients treated with liposomal irinotecan.

The labeling for liposomal irinotecan includes a boxed warning to alert health-care professionals about the risks of severe neutropenia and diarrhea. The drug is not approved for use as a single agent for the treatment of patients with metastatic pancreatic cancer. 

According to the National Cancer Institute, there will be 48,960 new cases of pancreatic cancer diagnosed in the United States in 2015 and nearly the same number of deaths caused by the disease (40,560). Pancreatic cancer can be difficult to diagnose early and treatment options are limited, especially when the disease has become metastatic and surgery to remove the tumor is not possible.   ■


1. Chen L-T, Von Hoff DD, Li C-P, et al: Expanded analyses of Napoli-1: Phase 3 study of MM-398 (nal-IRI), with or without 5-fluorouracil and leucovorin, versus 5-fluorouracil and leucovorin in metastatic pancreatic cancer previously treated with gemcitabine-based therapy. 2015 Gastrointestinal Cancers Symposium. Abstract 234. Presented January 15, 2015.