In early 2016, the FDA approved several important new cancer and hematology treatments.
cabozantinib (Cabometyx) was approved for advanced kidney cancer after prior therapy. In trials, it nearly doubled the time patients lived without disease progression compared to everolimus (7.4 vs. 3.8 months) and showed a median overall survival of 21.4 months. Common side effects included diarrhea and fatigue.
venetoclax (Venclexta) was approved for chronic lymphocytic leukemia with a 17p deletion. It works by blocking the BCL-2 protein to restore cancer cell death. About 80% of patients responded to treatment, achieving remission.
A new blood test called Epi proColon was also approved for colorectal cancer screening in people who refuse standard screening methods, though it still requires follow-up colonoscopy if positive.
Finally, defibrotide (Defitelio) was approved for a rare liver complication after stem cell transplant, with survival rates of 38–45% at 100 days.