FDA Grants Regular Approval to Venclexta Combo for Untreated Acute Myeloid Leukemia

The Food and Drug Administration (FDA) approved Venclexta (venetoclax) in combination with Vidaza (azacytidine), Dacogen (decitabine), or low dose cytarabine (LDAC) to treat patients with newly diagnosed acute myeloid leukemia (AML), aged 75 or older, who have comorbidities that exclude them from intensive induction chemotherapy, according to the agency.

"Today’s full approval is supported by the significant results that showed that Venclexta in combination with azacitidine extended overall survival for people with newly diagnosed acute myeloid leukemia who cannot tolerate intensive induction chemotherapy," said Dr. Levi Garraway, chief medical officer and head of Global Product Development at Genentech, in a statement to CURE®.

Venclexta was originally granted accelerated approval by the FDA in November 2018 and, therefore, needed efficacy to be confirmed in further trials for full approval by the agency. The FDA based full approval on two trials, VIALE-A and VIALE-C.

In the randomized, double-blind, placebo-controlled VIALE-A trial, researchers evaluated Venclexta in combination with Vidaza (286 patients) or placebo in combination with Vidaza (145 patients). The agency-based efficacy results on improved overall survival.

The addition of Venclexta improved median overall survival, compared with placebo (14.7 months versus 9.6 months), reducing the risk for death by 34%. Moreover, 37% of those in the Venclexta treatment arm showed a complete remission, compared with just 18% in the placebo arm.

In the randomized, double-blind, placebo-controlled VIALE-C trial, researchers evaluated Venclexta in combination with LDAC (143 patients, compared with placebo in combination with LDAC (68 patients). The FDA based efficacy results from this trial on complete remission rates and the duration of complete remission.

The addition of Venclexta, compared with placebo, demonstrated superior complete remission rates (27% versus 7.4%). Moreover, the agent extended median duration of complete remission (11.1 months versus 8.3 months, respectively). However, in this trial, Venclexta did not significantly improve overall survival compared with placebo.

The most common side effects included nausea, diarrhea, thrombocytopenia, constipation, neutropenia, febrile neutropenia, fatigue, vomiting, edema, pyrexia, pneumonia, dyspnea, hemorrhage, anemia, rash, abdominal pain, sepsis, musculoskeletal pain, dizziness, cough, oropharyngeal pain and hypotension.

Of note, review for full approval of the Venclexta combination was conducted under the FDA’s Project Orbis, where the agency collaborated with the Australian Therapeutic Goods Administration, the Brazilian Health Regulatory Agency, Health Canada and Switzerland’s Swissmedic.

To learn more about this approval, check back later on what this means for those with AML.

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