Wednesday, Dec 12, 2001

Genentech Receives FDA Approval to Add Survival Data to Herceptin Product Labeling

South San Francisco, Calif. -- December 12, 2001 --

Genentech, Inc. (NYSE: DNA) today announced that the U.S. Food and Drug Administration (FDA) has approved the inclusion of median survival data into the package insert of Herceptin® (Trastuzumab). The new labeling references the 24 percent increase in median overall survival for women with HER2 (human epidermal growth factor receptor2) positive metastatic breast cancer treated initially with Herceptin and chemotherapy compared to chemotherapy alone (median 25.1 months compared to 20.3 months). Genentech submitted a supplemental Biological License Application (sBLA) to the FDA for the label change in January 2001.

"Herceptin is the first targeted selective therapy for HER2 positive breast cancer and one of the few therapies that has demonstrated an increase in overall survival in metastatic breast cancer," said Gwen Fyfe, M.D., Genentech's senior director of Oncology, Medical Affairs. "When receiving an initial weekly treatment regimen of Herceptin and chemotherapy until disease progression, women with HER2 positive metastatic breast cancer may have a better chance for increased survival."

"This survival benefit shown with Herceptin not only is a significant advance in the treatment of breast cancer, but it also heralds the major transformation being made in the war on cancer by the use of targeted therapies," said Dennis J. Slamon, M.D., Ph.D., Chief of the Division of Hematology and Oncology at UCLA's Jonsson Comprehensive Cancer Center. "Herceptin represents a significant transition from a one-size-fits all approach to one based on finding ways to treat directed at the underlying molecular genetics of the disease. The survival benefit demonstrated by Herceptin represents one of the largest improvements made in the treatment of metastatic breast cancer in over four decades." Slamon's laboratory research aided in the development of Herceptin.

Survival Data

Final results from a pivotal Phase III trial of 469 women showed that those treated weekly until disease progression with Herceptin and standard cycles of chemotherapy (either anthracycline and cyclophosphamide or paclitaxel) as first-line treatment lived almost five months longer (median 25.1 months compared to 20.3 months) than women treated with either chemotherapy regimen alone.

Data from this trial also showed an increase in efficacy with Herceptin in both Herceptin plus chemotherapy subgroups. The combination of Herceptin plus chemotherapy compared to chemotherapy alone improved the overall response rate from 29 percent to 45 percent, prolonged the time to disease progression (TTP) from 4.5 to 7.2 months and increased median duration of response from 5.8 to 8.3 months.

HER2 Positive Disease

In approximately 25 percent of women with metastatic breast cancer, there is a genetic alteration in the HER2 gene that produces an increased amount of the growth factor receptor protein on the tumor cell surface. When compared to non-HER2 breast cancer, women whose tumors overexpress the HER2 protein are likely to have a more aggressive type of breast cancer with a poorer prognosis, shorter time to disease progression, increased relapse rate, shortened survival and disease that is not as responsive to standard therapies, including certain chemotherapy regimens.

HER2 Test Receives Positive Recommendation by FDA's Oncologic Drug Advisory Committee (ODAC)

On December 5th, the FDA's ODAC panel unanimously recommended approval of a supplemental Biologics License Application (sBLA) for the inclusion of fluorescence in situ hybridization (FISH) testing, by PathVysion(TM) (Vysis, Inc.), as an appropriate method to determine a patient's HER2 gene amplification level.

The recommendation for approval was based on data from an exploratory, prospectively-designed analysis demonstrating that FISH testing is reliable, accurate and has a high rate of concordance or agreement (88%) with the protein test used in Herceptin pivotal studies to identify HER2-positive breast cancer. Genentech will work with the FDA to determine next steps and pending approval, adding the use of the FISH test to the Herceptin package insert.

About Herceptin

Herceptin received FDA approval in September 1998 for use in women with metastatic breast cancer who have tumors that overexpress the HER2 protein. It is indicated for treatment of patients both as first-line therapy in combination with paclitaxel and as a single agent in second- and third-line therapy. Herceptin is marketed in the United States by Genentech and internationally by F. Hoffmann-LaRoche.

Herceptin generally is well-tolerated. Overall, the most common adverse events in the pivotal trial were fever, hematologic toxicity and cardiac dysfunction. Infusion-associated signs and symptoms were observed but did not require antibody discontinuation. In the post-marketing setting, rare serious and fatal infusion-related events have occurred. Most patients who experienced the more serious and fatal infusion-related events had pre-existing pulmonary compromise and advanced malignancies.

The most serious adverse event in the pivotal trial was cardiac dysfunction (NYHA Grade I-IV), which occurred more frequently when Herceptin was administered with anthracycline and cyclophosphamide (28 percent) compared with anthracycline and cyclophosphamide alone (7 percent), or with Herceptin plus paclitaxel (11 percent) compared to paclitaxel alone (1 percent). Although the NYHA Grade III/IV toxicity, which occurred in a smaller percentage of patients, was potentially severe and/or life threatening, signs and symptoms improved in 75 percent of patients.

Breast Cancer

Approximately 1.6 million women have been diagnosed with breast cancer in the United States. According to the American Cancer Society, 180,000 new cases are diagnosed in any given year. Genentech estimates there are approximately 164,000 women with metastatic breast cancer.


Genentech, Inc. is a leading biotechnology company that discovers, develops, manufactures, and markets human pharmaceuticals for significant unmet medical needs. Fifteen of the currently approved biotechnology products stem from or are based on Genentech science. Genentech manufactures and markets ten biotechnology products directly in the United States. The company has headquarters in South San Francisco, California, and is traded on the New York Stock Exchange under the symbol DNA.

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