On August 17th 2011, Roche and Daiichi Sankyo announced that the FDA approved Zelboraf and companion diagnostic for BRAF-mutation positive metastatic melanoma. Beyond the fact that this is probably one of the fastest FDA drug review and approval in recent history (3 months from submission to approval!), it is also a remarkable example of a drug being developed specifically in response to disease biomarker evidences.
The mutant form of the BRAF protein V600E, which shows increased signaling activity, has been identified as a potential prognostic or therapeutic response biomarker in multiple forms of advanced cancers (colorectal, thyroid, melanoma). In particular, the mutated form of BRAF is found in the 30% to 60% of melanomas in which it is thought to play a critical role in the malignancy process. Based on the thorough validation of this target in this type of cancer, Plexxikon Pharmaceuticals initiated a drug development program specifically aimed at the mutant form of BRAF. Early in 2011, the company released the results of their phase 3 study showing conclusive therapeutic effect of the PLX4032 molecule in previously untreated melanoma patients positive for the BRAF V600E mutation. Of note, because of the specificity of PLX4032, the study only enrolled patients with tumor positive for the mutated form of BRAF. Considering the high mortality rate of patients suffering from advanced melanoma, the approval of this new personalized treatment is expected to fill a critical unmet medical need.
Notes: In January 2011, Plexxikon entered an agreement with Roche – Genentech to co-promote PLX4032 in the US. In late February 2011, Plexxikon was acquired by Daiichi Sankyo.
Thierry
Sornasse for Integrated Biomarker Strategy
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