The U.S. Food and Drug Administration (FDA) has approved irinotecan liposome injection (Onivyde), in combination with fluorouracil (5-FU) and leucovorin, for the treatment of patients with advanced (metastatic) pancreatic cancer who have been previously treated with gemcitabine-based chemotherapy.1

The FDA had previously granted priority review and orphan drug designation for the liposomal formulation of irinotecan because, if approved, it would provide a significant improvement in effectiveness and safety in the treatment for advanced pancreatic cancer, for which limited treatment options currently exist, particularly for those with poor performance status.

“Many FDA staff who review drug applications are clinicians as well, so it’s especially rewarding when we are able to expedite access to new treatments for patients with unmet needs,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

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“By using the priority review designation for the application for Onivyde, patients will have earlier access to a drug that helps extend survival.”

Approval was based on the findings of a 3-arm, open-label study that evaluated liposomal irinotecan plus 5-FU/leucovorin compared with liposomal irinotecan alone and 5-FU/leucovorin alone in 417 patients with metastatic pancreatic adenocarcinoma who had experienced disease progression following treatment with gemcitabine or gemcitabine-based therapy.

Results showed that the addition of liposomal irinotecan to 5-FU/leucovorin improved overall survival by nearly 2 months compared with 5-FU/leucovorin alone. Progression-free survival was also 1.6 months longer with liposomal irinotecan than with 5-FU/leucovorin alone.

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In regard to safety, the most common side effects of treatment with liposomal irinotecan are diarrhea, fatigue, vomiting, nausea, decreased appetite, stomatitis, and pyrexia. The drug may also cause lymphopenia and neutropenia in some patients and sepsis-related death following neutropenia has been reported in patients treated with the drug.

Clinicians should also be aware of the boxed warning alerting health care professionals about the risks of diarrhea and severe neutropenia.

The American Cancer Society estimates that 48,960 people in the United States will be diagnosed with pancreatic cancer in 2015 and 40,560 people will die from the disease.2 Pancreatic cancer accounts for about 3% of all cancer in the United States. Patients with stage 4 metastatic disease only have about a 1% 5-year survival rate.


  1. FDA approves new treatment for advanced pancreatic cancer [news release]. Bethesda, MD: U.S. Food and Drug Administration; October 22, 2015. PressAnnouncements/ucm468654.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed October 22, 2015. American Cancer Society. Cancer facts & figures 2015. Atlanta, GA: American Cancer Society; 2015.