Pancreatic cancer can be difficult to diagnose early and treatment options are limited, especially when the disease has metastasized; surgery to remove the tumor is not possible.
“This is an important day for patients facing pancreatic cancer,” said Andrea Wang-Gillam, MD, PhD, an associate professor of medicine and the clinical director of the GI oncology program, Division of Oncology, at Washington University School of Medicine, in St. Louis, and a key investigator on the Onivyde Phase 3 NAPOLI-1 study. “With a long history of failed clinical studies in the post-gemcitabine setting, this approval is a significant achievement in the oncology community. It brings a new therapy to the many patients who are facing this aggressive disease and are in need of treatment options.”
The FDA granted priority review and orphan drug designations for Onivyde, which is novel encapsulation of irinotecan in a long-circulating liposome. 
The approval was based on the results of an international Phase III, randomized controlled study (NAPOLI-1), in which irinotecan liposome injection in combination with 5-FU and leucovorin achieved its primary end point of a significant improvement in overall survival (OS) (unstratified hazard ratio [HR], 0.68; 95% CI, 0.50-0.93; P=0.014) with a 45% improvement in median overall survival of 6.1 months for patients receiving the Onivyde combination regimen compared with 4.2 months for patients who received 5-FU and leucovorin alone. The combination treatment also demonstrated improvement in progression-free survival (PFS) (3.1 vs. 1.5 months; HR, 0.55; 95% CI, 0.41-0.75). The monotherapy regimen in this study did not achieve its primary end point, and therefore Onivyde is not indicated as a single agent.
Approximately 49,000 individuals are diagnosed with pancreatic cancer each year in the United States, the overwhelming majority of whom have adenocarcinoma. Most patients receive gemcitabine-based therapy during either adjuvant/neoadjuvant treatment for locally advanced disease or first- or second-line therapy for metastatic disease, but are left with no standard-of-care therapy upon progression. Onivyde in combination with 5-FU and leucovorin is now approved for these patients whose disease has progressed following gemcitabine-based therapy.
The most common adverse events of combination treatment were diarrhea, fatigue/asthenia, vomiting, nausea, decreased appetite, stomatitis and pyrexia, and the most common severe laboratory abnormalities were lymphopenia and neutropenia. The labeling for Onivyde includes a boxed warning to alert health care professionals about the risks for severe neutropenia and diarrhea. Onivyde is not approved for use as a single agent for the treatment of patients with metastatic pancreatic cancer.
“This is a pivotal achievement for the pancreatic cancer community because it provides a new treatment option for some patients facing this difficult disease,” said Julie Fleshman, president and CEO of the Pancreatic Cancer Action Network. “We applaud the dedication of those involved in this advancement, knowing it will impact our goal to double pancreatic cancer survival by 2020.”