FDA Approves Zelboraf for Treatment of Metastatic Melanoma

Strategic Insights -

The US Food and Drug Administration (FDA) has approved Zelboraf (vemurafenib) for the treatment of inoperable or metastatic melanoma with a BRAF V600E mutation, as determined by an FDA-approved test. This approval marks a significant milestone in the treatment of metastatic melanoma, a devastating disease with limited approved treatment options until now. Zelboraf is the first and only FDA-approved personalized medicine shown to improve survival in people with BRAF V600E mutation-positive metastatic melanoma.

Key Takeaways:

  • The FDA approval of Zelboraf is based on results from two clinical studies (BRIM3 and BRIM2) in people with BRAF V600E mutation-positive, inoperable or metastatic melanoma.
  • Zelboraf has been shown to improve overall survival (OS) and investigator-assessed progression-free survival (PFS) in patients with previously untreated BRAF V600E mutation-positive, unresectable or metastatic melanoma.
  • The confirmed investigator-assessed response rate in people who received Zelboraf was 48.4 percent, compared to 5.5 percent for those who received chemotherapy.
  • Zelboraf may cause a type of skin cancer called cutaneous squamous cell carcinoma (cuSCC), and patients should be monitored for skin changes and avoid going out in the sun while taking the medication.
  • The BRAF protein is a key component of the RAS-RAF pathway involved in normal cell growth and survival, and mutations that keep the BRAF protein in an active state may cause excessive signaling in the pathway, leading to uncontrolled cell growth and survival.
  • The cobas 4800 BRAF V600 Mutation Test is a polymerase chain reaction-based diagnostic test developed by Roche, which is clinically valid to identify tumors that carry the BRAF V600E mutation.
  • Zelboraf is an oral, small molecule, kinase inhibitor indicated for the treatment of patients with unresectable or metastatic melanoma with BRAF V600E mutation as detected by an FDA-approved test.

Statistics:

  • The BRAF protein is thought to occur in an estimated half of all melanomas and eight percent of solid tumors.
  • The American Cancer Society estimates there will be more than 70,000 new cases of melanoma and nearly 8,800 melanoma deaths this year in the United States.
  • The confirmed investigator-assessed response rate in people who received Zelboraf was 48.4 percent.
  • Zelboraf has been shown to reduce the risk of death by 56 percent and the risk of the disease getting worse or dying by 74 percent in patients with previously untreated BRAF V600E mutation-positive, unresectable or metastatic melanoma.

Sources:

  • Genentech. (2011). FDA Approves Zelboraf for Treatment of Metastatic Melanoma.
  • US Food and Drug Administration. (2011). FDA Approves Zelboraf for Metastatic Melanoma.
  • American Cancer Society. (2011). Cancer Facts and Statistics.
  • Roche. (2011). cobas 4800 BRAF V600 Mutation Test.