ROCKVILLE, Md--Because advanced pancreatic cancer is a devastating disease with no effective treatment, members of the FDA Oncologic Drugs Advisory Committee (ODAC) voted to recommend approval of the nucleoside analog Gemzar (gemcitabine hydrochloride, Eli Lilly) as a first-line treatment for patients with nonresectable stage II, stage III, or metastatic (stage IV) adenocarcinoma of the pancreas.
The agent is currently available under a treatment investigational new drug program for selected patients with advanced or metastatic pancreatic cancer, which will continue while the company awaits the FDA's final decision on approval.
In the United States, 27,000 people die each year of pancreatic cancer, usually less than a year after diagnosis. "Previous clinical trials have shown that fluorouracil(Drug information on fluorouracil), the most commonly used treatment for pancreatic cancer, in combination with other chemotherapeutic agents is no better than fluorouracil alone," said Daniel Von Hoff, MD, Cancer Therapy and Research Center, San Antonio. Therefore, fluorouracil was the drug against which Gemzar was tested.
Lilly presented two clinical trials: a phase III randomized, blinded (but not double blind) study comparing Gemzar with fluorouracil, and a phase II single-arm study of patients whose disease had progressed while they were receiving fluorouracil.
New Surrogate Endpoint
Dr. Von Hoff noted that it is difficult to evaluate advanced pancreatic cancer patients following surgery, radiation, and chemotherapy using traditional endpoints such as the rate of tumor growth or shrinkage, since these often do not provide a meaningful analysis of a patient's overall health.
"Because clinical endpoints are so hard to measure in this disease, we wanted to set up a trial to determine the effect of Gemzar on what bothers patients most," he said.