Last week, Novartis announced that its combination therapy Tafinlar (dabrafenib) + Mekinist (trametinib) was granted Priority Review by the U.S. Food and Drug Administration (FDA) for the adjuvant treatment of stage 3 BRAF V600 mutation-positive melanoma.
In October, the FDA granted Breakthrough Therapy designation to the combination therapy. Its original approval was for patients with unresectable or metastatic melanoma with a BRAF V600E/K mutation as detected by an FDA-approved test and non-small cell lung cancer (NSCLC) with a BRAF V600E mutation, which came in November 2015
Tafinlar is a BRAF inhibitor, while Mekinist is an MEK 1/2 inhibitor, and the new designation is based on the combination therapy meeting its primary endpoints in 870 patients with stage 3 BRA V600E/K mutation-positive melanoma who had undergone complete resection in the COMBI-AD phase 3 study. Tafinlar + Mekinist significantly reduced the risk of disease recurrence or death by 53% when compared to placebo.
Key secondary endpoints were also met in the study, and the combination treatment group also exhibited improvement in overall survival (OS), remaining consistent with the efficacy profile of the therapy, which have shown its ability to significantly improve OS when compared to monotherapies. The safety and efficacy of the combination, however, have not been established outside of the approved indication.
"The FDA's decision to grant Tafinlar in combination with Mekinist Breakthrough Therapy designation and Priority Review designation validates the potential of the combination to have a significant impact on the lives of melanoma patients treated in the adjuvant setting," said Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development in a press release
. "There remains a need to address the high risk of recurrence seen in these patients and improve the quality of care they receive."
All variations of melanoma – a cancer in which malignant cells form in the melanocytes, or cells that color the skin – are considered “rare,” and the American Cancer Society estimates that nearly 90,000 new melanomas will be diagnosed in 2017 with almost 10,000 people expected to die from the disease.
The regulatory action came on the same day that Novartis announced the FDA approval
of the inclusion of treatment-free remission (TFR) data for Tasigna (nilotinib) in Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP).
For more from the FDA, including applications, designations and approvals, follow Rare Disease Report