Nanotherapeutic Approved for Advanced Pancreatic Cancer

The U.S. Food and Drug Administration approval provides a new option for patients who face a poor prognosis

Onivyde, a nanotherapeutic form of the conventional chemotherapeutic irinotecan, has been approved by the U.S. Food and Drug Administration (FDA) for treating certain patients with pancreatic cancer.

The new nanotherapeutic is approved for use in combination with two other conventional chemotherapy drugs, fluorouracil and leucovorin, and the three-drug combination is intended for patients with advanced pancreatic cancer that has progressed despite treatment with gemcitabine-based chemotherapy. The nanotherapeutic-containing drug combination was shown to extend overall survival for patients in a randomized phase III clinical trial.

This progress is particularly welcome given that advanced pancreatic cancer is one of the most deadly forms of cancer; just 2 percent of patients to receive this diagnosis survive five years.

Nanotechnology is defined by the National Institutes of Health as the understanding and control of matter at dimensions of about 1 to 100 nanometers; one nanometer is one-billionth of a meter. To provide perspective on just how small this is, the thickness of a sheet of paper is about 100,000 nanometers, as is the diameter of a strand of human hair.

Anticancer nanotherapeutics comprise an anticancer agent and a nanosized carrier that can help transport the anticancer agent to the patient’s tumors, protect the anticancer agent from degradation, and increase the length of time the anticancer agent is active in the body, among other things. As a result, anticancer nanotherapeutics allow the delivery of higher levels of anticancer agents to tumors than traditional systemic delivery methods, increasing effectiveness while reducing toxicity in noncancerous tissues.

In the case of Onivyde (also called irinotecan liposome injection and previously referred to as MM-398), the anticancer agent is the conventional chemotherapeutic irinotecan, which is used to treat metastatic colorectal cancer, and the nanosized carrier is a liposomal sphere.

The FDA approval of the nanoliposomal formulation of irinotecan was based on results from a phase III clinical trial called NAPOLI-1. Specifically, it was based on the demonstration that adding nanoliposomal irinotecan to fluorouracil and leucovorin extended median overall survival for patients with metastatic pancreatic adenocarcinoma whose cancer had grown after receiving the chemotherapeutic gemcitabine or a gemcitabine-based therapy by 1.9 months, from 4.2 months to 6.1 months.

Clinical trials testing whether the nanoliposomal formulation of irinotecan might also be effective against breast cancer and pediatric sarcoma are underway, and we look forward to learning whether this new anticancer nanotherapeutic will one day benefit patients with these diseases.

The FDA approval was rendered on Oct. 22, 2015.