New Drug Applications Submitted in the United States and Europe for Vemurafenib in Advanced Skin Cancer

Vemurafenib is the First Personalized Investigational Medicine to Have Shown a Significant Overall Survival Benefit in Metastatic Melanoma

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the company submitted a New Drug Application for vemurafenib (RG7204, PLX4032) to the U.S. Food and Drug Administration (FDA) for people with BRAF V600 mutation-positive metastatic melanoma, and that Roche submitted a Marketing Authorization Application to the European Medicines Agency (EMA) for vemurafenib in the same indication. Roche also submitted an application for the cobas 4800 BRAF V600 Mutation Test, a companion diagnostic. Vemurafenib, a “BRAF-inhibitor,” is designed to selectively target and inhibit a mutated form of the BRAF protein found in about half of all cases of melanoma, the deadliest and most aggressive form of skin cancer.

“We have worked swiftly to advance the vemurafenib development program knowing that patients with metastatic melanoma have a poor prognosis and limited options,” said Hal Barron, M.D., chief medical officer and head, Global Product Development. “The regulatory submissions of vemurafenib and the companion diagnostic to identify people with the type of melanoma specifically targeted by this medicine are exciting steps toward our goal of delivering a personalized therapy for this disease.”

The submissions are based on results from two positive clinical studies (BRIM2 and BRIM3) that evaluated vemurafenib in people with BRAF V600 mutation-positive metastatic melanoma, as determined by the investigational companion diagnostic test also being developed by Roche. Roche submitted a Premarket Approval Application (PMA) for the cobas 4800 BRAF V600 Mutation Test in the U.S. The test will also be registered in Europe.

About Metastatic Melanoma and BRAF

When melanoma is diagnosed early, it is generally a curable disease. However, when it spreads to other parts of the body, it is the deadliest and most aggressive form of skin cancer. A person with metastatic melanoma typically has a short life expectancy that is measured in months. The American Cancer Society estimates there were almost 70,000 new cases of melanoma and more than 8,000 melanoma deaths last year in the U.S. alone.

The BRAF protein is a key component of the RAS-RAF pathway involved in normal cell growth and survival. Mutations that keep the BRAF protein in an active state may cause excessive signaling in the pathway, leading to uncontrolled cell growth and survival. These mutations of the BRAF protein are thought to occur in an estimated half of all melanomas and eight percent of solid tumors.

About BRIM3 and BRIM2

BRIM3 is a global, randomized, open-label, controlled, multicenter, Phase III study that compared vemurafenib to dacarbazine chemotherapy, a current standard of care, in 675 patients with previously untreated BRAF V600 mutation-positive, unresected or locally advanced metastatic melanoma. The study met its co-primary endpoints and showed that participants who received vemurafenib lived longer (overall survival) and also lived longer without their disease getting worse (progression-free survival or PFS) compared to those who received dacarbazine chemotherapy. The safety profile was consistent with previous vemurafenib studies.

Full data from BRIM3 will be presented at the 47th Annual Meeting of the American Society of Clinical Oncology (ASCO) on June 5, 2011 in Chicago.

BRIM2 is a global, single-arm, multicenter, open-label Phase II study that enrolled 132 patients with previously treated BRAF V600 mutation-positive metastatic melanoma. The primary endpoint of the study was overall response rate as assessed by an independent review committee. The study showed that vemurafenib shrank tumors in 52 percent of trial participants. People who participated in the trial lived a median of 6.2 months without their disease getting worse (median PFS). Updated data from BRIM2 will also be presented at the ASCO Annual Meeting.

The most frequent Grade 3 adverse event observed in clinical trials of vemurafenib was cutaneous squamous cell carcinoma, a common skin cancer treated by local excision (minor surgery done in a physician’s office). The most common adverse events were rash, increased sun sensitivity, joint pain, hair loss and fatigue. Possible serious side effects of vemurafenib include liver problems, changes in heartbeat or very fast or abnormal heartbeats and allergic reactions.

About Vemurafenib

Vemurafenib (pronounced vem yoo RAF en ib) is an investigational, oral, small molecule that is designed to selectively inhibit a cancer-driving mutated form of the BRAF protein. Vemurafenib is being co-developed under a 2006 license and collaboration agreement between Roche/Genentech and Plexxikon. The cobas 4800 BRAF V600 Mutation Test is an investigational, polymerase chain reaction-based companion diagnostic being developed by Roche to identify people whose tumors carry the BRAF V600 mutation.

Genentech is considering a broad development program with vemurafenib that may include combinations with other medicines (both approved and investigational, from Genentech and other companies), as well as studies in other tumor types. While Genentech/Roche seeks approval of vemurafenib, vemurafenib is available to eligible patients with BRAF V600 mutation-positive metastatic melanoma through a global patient access program. More information about this program or other vemurafenib studies is available at (in the U.S.) or the Roche Clinical Trials Registry at (in the EU). Genentech can also be contacted by calling the company’s clinical trial call center at 888-662-6728 or emailing [email protected].

About Genentech

Founded more than 30 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit


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