US FDA approves Vemurafenib , a Melanoma Drug

The United States Food and Drug Administration has approved vemurafenib (Zelboraf), a drug designed to treat patients with metastatic melanoma. Vemurafenib is a BRAF-inhibitor and is a personalized investigational drug intended to treat patients who test positive for the BRAF mutation. It acts by particularly restricting the mutant BRAF-protein activity seen in nearly 50% of melanoma cases (the most aggressive and fatal form of skin cancer).

Director of the Donald A. Adam Comprehensive Melanoma Research Center at Moffitt as well as trial researcher, Jeffrey Weber, M.D., Ph.D., said: “This is a great success story. To find a mutation and develop a test to enable widespread testing and matching a patient to the treatment is just the kind of personalized medicine we want to be known for here at the cancer center.”

The Phase II, open-label, single-arm, multicenter global trial investigated the effects of administering BRIM2 to 132 patients with BRAF V600 mutation-positive metastatic melanoma. The overall response rate was the primary endpoint of the study, which was assessed by an independent review committee who reported that vemurafenib decreased tumors in 52% of trial participants. These participants went on to spend a median of 6.2 months without disease progression (median PFS).

The most common Grade 3 adverse event was the occurrence of cutaneous squamous cell carcinoma, which was excised by a minor surgery performed at the physician’s office. Joint pain, rash, increased sun sensitivity, hair loss, and fatigue were the recurrent adverse events. Changes in heartbeat, very fast or abnormal heartbeats, liver problems, and allergic reactions were amongst the potential serious side effects with vemurafenib administration.