Novartis’ combination therapy Tafinlar + Mekinist has won approval from the US Food and Drug Administration to treat patients with metastatic non-small cell lung cancer (NSCLC) carrying the BRAF V600E mutation.

Clearance comes on the back of safety and efficacy data from a Phase II, three-cohort, multicenter, non-randomised and open-label study including patients with late-stage disease, which also formed the basis of European approval in March.

In the trial, 36 treatment-naïve patients receiving 150mg of Tafinlar twice daily and 2mg of Mekinist once daily demonstrated an objective response rate of 61 percent. In this population, 68 percent of patients had not progressed after nine months.

In the previously treated population receiving the same dosage, patients demonstrated an ORR of 66.7 percent. The response was durable with a median DoR reaching 9.8 months, the from noted.

The most common adverse events with an incidence of more than 20 percent were pyrexia, nausea, vomiting, peripheral oedema, diarrhoea, dry skin, decreased appetite, asthenia, chills, cough, fatigue, rash, and dyspnoea.

BRAF mutations occur in around 1-3 percent of NSCLC cases worldwide. Novartis says there is an urgency to treat people with this mutation, as BRAF V600E mutation-positive tumours have been shown to be more aggressive and may lead to a poorer prognosis.

"Patients with BRAF V600E mutation-positive metastatic NSCLC have responded less favourably to standard chemotherapy, suggesting that there is a critical need for a targeted therapy," said Bruno Strigini, chief executive of Novartis Oncology.

The approval of the Tafinlar + Mekinist combination “validates our expertise in tumor biology, which enabled us to develop the first targeted treatment for people with this rare mutation."