Authors' objectives: Vemurafenib (Zelboraf) is intended for the treatment of BRAF V600 mutation positive, metastatic melanoma in paediatric patients. If licensed, vemurafenib will offer an additional oral treatment option for the subset of paediatric patients with BRAF V600 mutation positive, metastatic melanoma, who currently have few effective specifically licensed therapies available. Vemurafenib is a low molecular weight, orally available inhibitor of BRAF serine-threonine kinase. Vemurafenib is licensed in the EU as monotherapy for the treatment of adult patients with BRAF V600 mutation positive, unresectable or metastatic melanoma. Over the last thirty years, rates of malignant melanoma in the UK have risen faster than any of the other current ten most common cancers. It is now the fifth most common cancer in England and is the most common skin cancer in children. Carcinomas of sites other than kidney, liver and gonads account for 4% of all childhood cancers of which 24% are malignant melanomas (an estimated 13 cases in England per year). At presentation, 10% of cutaneous melanomas will have metastasised. Approximately 50% of melanomas harbour activating BRAF mutations and over 90% of these are BRAF V600 mutations. Non-surgical modalities including immunotherapy, chemotherapy, radiation therapy, or a combination of these treatments may be offered for inoperable stage III or IV disease, however many current systemic therapies are not currently licensed for use in children and adolescents. Vemurafenib is currently in a phase I clinical trial evaluating dose regimens and pharmacokinetics in patients aged 12 to 17 years with unresectable stage IIIC or IV melanoma. The trial is expected to complete in February 2017.

Project Status: Completed

Year Published: 2015

URL for published report: www.hsric.nihr.ac.uk/topics/vemurafenib-zelboraf-for-metastatic-braf-v600-mutation-positive-melanoma-in-children-and-adolescents/

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Horizon Scanning Centre

Contact Address: The NIHR Horizon Scanning Centre, Department of Public Health, Epidemiology, and Biostatistics, School of Health and Population Sciences, University of Birmingham, 90 Vincent Drive, Edgbaston, Birmingham, B15 2SP. United Kingdom. Tel: +44 121 414 7831, Fax: +44 121 2269

Contact Name: c.packer@bham.ac.uk

Contact Email: c.packer@bham.ac.uk

Copyright: NIHR Horizon Scanning Research&Intelligence Centre (NIHR HSRIC)