Two New Therapies Approved by FDA for Advanced Kidney Cancer

June 3, 2016, by NCI Staff

The Food and Drug Administration (FDA) recently approved two drugs for the treatment of patients with advanced kidney cancer. Both approvals are for patients whose cancers have progressed after receiving prior treatment with drugs that block tumor blood vessel growth, known as antiangiogenic therapies.

On April 15, the agency approved cabozantinib (Cabometyx™), and on May 13 it approved lenvatinib (Lenvima®) in combination with everolimus (Afinitor®).

Both cabozantinib and lenvatinib are multi-targeted agents that simultaneously inhibit the activity of several molecular targets in cancer cells. Both drugs block the activity of proteins involved in angiogenesis and in other functions that promote tumor cell survival and growth.

Cabozantinib’s approval was based on the results of a phase III randomized clinical trial comparing the drug with everolimus, which the FDA approved in 2009 for patients with kidney cancer whose disease has progressed after prior treatment. In the trial, patients treated with cabozantinib had longer median progression-free survival than patients treated with everolimus (7.4 months versus 3.8 months) and longer median overall survival (21.4 months versus 16.5 months). Seventeen percent of patients treated with cabozantinib experienced a confirmed response to treatment, compared with 3% of patients treated with everolimus.

Lenvatinib’s approval was based on the results of a randomized phase II clinical trial. The trial had three treatment arms: lenvatinib in combination with everolimus, lenvatinib alone (but at a higher dose than in the combination therapy arm), and everolimus alone.

Patients treated with lenvatinib plus everolimus had a median progression-free survival of 14.6 months versus 5.5 months for those treated with everolimus alone. According to the FDA, updated data from the trial showed a trend toward improved overall survival in patients who received lenvatinib plus everolimus, although the trend did not reach statistical significance.

Common side effects of both cabozantinib and lenvatinib included fatigue, nausea, diarrhea, and hypertension; serious adverse events were common in both trials.

Among patients treated with cabozantinib, 60% had to have their dose decreased during the trial. And for lenvatinib, nearly 20% of patients in the trial had high-grade diarrhea, prompting the agency to include a special warning on the drug’s label about the risk of this side effect.