The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to GlaxoSmithKline's Tafinlar (dabrafenib) for treatment of patients with metastatic BRAF V600E mutation-positive non-small cell lung cancer (NSCLC) who have received at least one prior line of platinum-containing chemotherapy.  Dabrafenib is not approved or licensed anywhere in the world for use in this treatment setting.

The Breakthrough Therapy designation was based on interim efficacy and safety results from an ongoing phase II study of dabrafenib administered orally to 25 patients who had NSCLC with the BRAF V600E mutation and who had received at least one previous course of chemotherapy. These interim results were presented at the 2013 American Society for Clinical Oncology Annual Meeting.

Lung cancer is the second most common cancer in both men and women and is by far the leading cause of cancer-related death worldwide. Recent advances in the understanding of tumour biology have identified genetic mutations, such as mutation in the BRAF protein, that can drive malignant cell growth and tumour proliferation in NSCLC. It is estimated that the BRAF V600E mutation targeted by dabrafenib is present in approximately 2.0% of patients with NSCLC.

The Breakthrough Therapy designation was enacted as part of the 2012 FDA Safety and Innovation Act and is intended to expedite development and review of drugs to treat serious or life-threatening medical conditions when preliminary clinical evidence demonstrates that the drug may have substantial improvement on at least one clinically significant endpoint over available therapies.  Breakthrough Therapy designation includes all the features of the Fast Track designation, as well as more intensive guidance from the FDA on a drug’s clinical development programme.