FDA approves Venclexta plus a hypomethylating agent or low-dose cytarabine, for the treatment of newly-diagnosed AML patients who are age 75 years or older or ineligible for intensive induction chemotherapy.- Genentech/Roche

Genentech, a member of the Roche Group announced that the FDA has granted accelerated approval to Venclexta (venetoclax), in combination with a hypomethylating agent (azacitidine or decitabine), or low-dose cytarabine (LDAC), for the treatment of people with newly-diagnosed acute myeloid leukemia (AML), who are age 75 years or older, or for those ineligible for intensive induction chemotherapy due to coexisting medical conditions. AML is the most common type of aggressive leukemia in adults and has the lowest survival rate for all types of leukemia. This accelerated approval was based on results from the M14-358 study and the M14-387 study in people newly-diagnosed with AML including those who were ineligible for intensive induction chemotherapy. In M14-358, the rate of complete remission (CR) was 37 percent (n=25/67) and the rate of complete remission with partial blood count recovery (CRh) was 24 percent (n=16/67) for those who received Venclexta plus azacitidine. For those who received Venclexta plus decitabine, the rate of CR was 54 percent (n=7/13) and the rate of CRh was 8 percent (n=1/13). M14-387 showed a CR rate of 21 percent (n=13/61) and a CRh rate of 21 percent (n=13/61) for those who received Venclexta in combination with LDAC. The most common serious side effects of these regimens (occurring in at least 5 percent of patients) were low white blood cell count with fever, pneumonia, bacteria in the blood, inflammation of tissue under the skin, device-related infection, diarrhea, fatigue, bleeding, localized infection, multiple organ dysfunction syndrome and respiratory failure.

The FDA's Accelerated Approval Program allows conditional approval of a medicine that fills an unmet medical need for a serious condition. This approval of Venclexta is based on surrogate endpoints that are reasonably likely to predict clinical benefit, including CR and CRh. Continued approval for this indication may be contingent upon verification and description of clinical benefit observed in confirmatory trials..