Mutation to Medicine

Improved Survival with Vemurafenib in Melanoma

Hensin Tsao, MD, PhD


Journal Watch 

In This Article


This landmark study may well prompt FDA activity. Wider access to vemurafenib would enlarge the melanoma medicine cabinet for late-stage disease. Despite the excitement, several points are worth noting: First, follow-up in the interim analysis was short, so longer-term data are needed. Second, for the many patients without BRAF V600E mutations, immunologic treatments and targets further downstream (e.g., MEK) may be an answer, but truly personalized medicine depends on having options for every genotype. Additionally, even some patients with BRAF V600E mutations will not respond, so markers of likely response would be beneficial. Most importantly, many patients with an initial response soon relapse: Vemurafenib delivers a concussive but not a lethal blow. Studies of vemurafenib resistance mechanisms have been published, and the pipeline for future analysis is open. If approved, vemurafenib will become the first-choice drug for many, but unmet needs will leave us all wanting more than the drug can deliver.


Comments on Medscape are moderated and should be professional in tone and on topic. You must declare any conflicts of interest related to your comments and responses. Please see our Commenting Guide for further information. We reserve the right to remove posts at our sole discretion.