FDA Grants Treatment IND for Lilly's Investigational Cancer Drug Gemzar - gemcitabine hydrochloride.

INDIANAPOLIS--(HealthWire)--February 21, 1995--Eli Lilly and Company has received authorization from the U.S. Food and Drug Administration (FDA) to make gemcitabine hydrochloride, an investigational anticancer agent for advanced pancreatic cancer, available through a Treatment Investigational New Drug (IND) program to patients who are not candidates for surgery. Gemcitabine is manufactured under the trade name Gemzar(R).

The FDA's Treatment IND regulations offer a mechanism that allows drug developers to provide earlier and wider access to investigational therapies for patients with immediately life-threatening or otherwise serious diseases for which there is no satisfactory alternative treatment. This occurs while review of a new drug application for the same indication is pending.

Gemcitabine will be available, free of charge, to patients who meet certain medical eligibility criteria. The drug will be available only through physicians who enroll in the Treatment IND program. Treatment with gemcitabine will follow a structured clinical protocol approved by the FDA. Physicians interested in entering patients in the Treatment IND program may call 1-800-621-7111 for more information.

Gemcitabine is not approved for marketing in the United States. The Treatment IND program will continue while a new drug application (NDA) to determine the safety and efficacy of gemcitabine in pancreatic cancer is under review by the FDA. The NDA was submitted to the FDA on February 1, 1995.

About 27,000 people in the United States were diagnosed with pancreatic cancer in 1994. Pancreatic cancer is generally asymptomatic until late in the course of the disease. It is among the most difficult cancers to treat and is rarely curable. The FDA authorized the Treatment IND for gemcitabine to provide a promising treatment for locally advanced or metastatic pancreatic cancer. Patients may have had either one prior chemotherapy regimen or none.

A preliminary review by FDA of two clinical trials of gemcitabine suggests that it may have some effectiveness in the treatment of this disease. Lilly carried out two trials in patients with pancreatic cancer, one a comparison with 5 fluorouracil (5-FU) in previously untreated patients, the other a study in 5-FU failures. The studies measured tumor shrinkage, survival, and an overall estimate of clinical benefit response rate. Clinical benefit response rate is a composite measure of symptomatic improvement, including assessments of pain, performance status (ability to perform daily activities), and weight.

An average of 7 percent of patients in both studies showed a partial response rate (a 50 percent or greater decrease in tumor size). There was a small (about one and a half month) improvement in median survival in the study comparing gemcitabine with 5-FU. In this study, patients treated with gemcitabine experienced an approximately 25 percent clinical benefit response rate compared with 5 percent of patients treated with 5-FU. The 1-year survival rates in the comparative trial were 18 percent and 2 percent for gemcitabine and 5-FU, respectively.

A second Phase II trial included patients who had not responded to 5-FU treatment. Symptoms improved in 27 percent of patients, with a median survival time of 3.85 months and a 1-year survival rate of 4 percent. A partial response rate was observed in approximately 10 percent of patients, and disease stabilization was reported in approximately 28 percent of patients.

The major side effects of gemcitabine included neutropenia, a decrease in white blood cells, which increases susceptibility to infection; thrombocytopenia, a decrease in blood platelets, which can cause excessive bleeding; and elevation of liver enzymes. Nausea, vomiting, rash, flu-like symptoms, breathing difficulties and traces of blood and protein in the urine have been reported. Rarely, hemolytic- uremic syndrome (anemia associated with kidney failure), was reported.

The Treatment IND program for gemcitabine is evidence of Lilly's ongoing commitment to improving the lives of cancer patients and is designed with a goal of further advancing the treatment of pancreatic cancer.

Lilly is a global research-based pharmaceutical corporation headquartered in Indianapolis, Indiana, that is working with its customers worldwide to help ensure that diseases are prevented, managed, and cured with maximum benefit and minimum cost to patients and society.

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Gemzar(R) (gemcitabine hydrochloride, Lilly)

CONTACT: Eli Lilly and Company, Indianapolis

Farnaz Khadem, 317/277-1172