Co-development of a companion diagnostic for targeted cancer therapy

Oncology drug development is a long and costly process associated with a success rate of 5–10%. The parallel development of companion diagnostic tests that will identify patients most likely to receive benefit has the potential to increase the success rate for oncology drugs and decrease development time and associated costs.Metastatic melanoma is a challenging disease that has been associated with poor survival. Identification of a mutated BRAF kinase gene in many cases of melanoma provided a promising therapeutic target. Here we describe the successful co-development of vemurafenib, a first-in-class selective inhibitor of oncogenic BRAF kinase, and its companion diagnostic, the cobas® 4800 BRAF V600 Mutation Test. Key success factors in the development process included early identification of the BRAF V600E biomarker, early development of the diagnostic test, and early and close collaboration between the pharmaceutical and diagnostic development teams. This focused and integrated process resulted in the first personalized medicine for the treatment of metastatic melanoma less than five years after the Investigational New Drug Application, a remarkably short time.

► Vemurafenib is a first-in-class selective inhibitor of oncogenic BRAF kinase.
► The cobas® 4800 BRAF V600 Mutation Test is the companion diagnostic for vemurafenib.
► Vemurafenib and the cobas® BRAF test were successfully developed in parallel.
► Co-development resulted in the first personalized medicine for metastatic melanoma.
► The time from IND to approval of drug and companion diagnostic was remarkably short.