Study demonstrates efficacy, tolerability of vemurafenib in aggressive form of metastatic melanoma

Open label safety study, published in The Lancet Oncology, shows impact of treatment in wide range of patient sub groups

The Lancet Oncology has published results from the largest ever trial of a single melanoma treatment. The open label safety study was conducted to assess the tolerability of Zelboraf^® (vemurafenib), and featured 3,222 patients from across 44 countries. 246 of these patients were from the UK.

The data reinforce the tolerability of vemurafenib – the first and only personalised treatment to extend life in patients with BRAF V600 mutation positive unresectable or metastatic melanoma – showing adverse events consistent with those reported in previous clinical trials. The trial also assessed the efficacy of the treatment, and demonstrated progression-free survival (PFS) of 5.6 months and overall survival (OS) of 12 months.

Dr James Larkin, Consultant Medical Oncologist at the Royal Marsden Hospital and lead investigator of this trial, said: “We are delighted to have been able to further explore the treatment options for patients with metastatic melanoma. Through this trial, we have been able to extend treatment with vemurafenib to ‘real world’ patients. This means that we can be confident that vemurafenib is a well-tolerated and potentially effective treatment for a broad group of patients, even those who previously would have been deemed unfit for a trial – for example patients with brain metastases. This is a huge step forward in the treatment of the disease.”

An extensive number of patient sub-groups were assessed in this landmark study, which included patients whose cancer has spread throughout the body to other organs, such as the brain. The data indicated that vemurafenib is well-tolerated in these patients, who are not typically evaluated in clinical trials due to the complexity of their condition. As the first study to provide a detailed and comprehensive analysis of the treatment’s impact on a breadth of patient populations at various stages of this disease, the data offer clinicians a strong foundation upon which they can tailor their treatment decisions.

Melanoma, which can be fatal if left untreated, is classified as metastatic when it has advanced beyond the skin to another part of the body. Until the availability of vemurafenib, people with the advanced form of this disease had very limited treatment options and their life expectancies were measured in months.

The incidence of melanoma is increasing, with nearly 13,000 new cases every year. Although metastatic melanoma typically has been associated with poor prognosis, with 5-year survival of about 60% and 15% in patients with regional and distant metastases, therapeutic options have recently changed substantially. The BRAF kinase inhibitor vemurafenib prolonged survival in patients with metastatic melanoma and is now approved and funded as a first-line treatment option in England and Scotland for patients with BRAF V600 mutation positive disease.