The U.S. Food and Drug Administration (FDA) approved venetoclax, in combination with obinutuzumab, for the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
The approval was based on results from the randomized, multicenter, open label CLL14 trial, which enrolled 432 patients with previously untreated CLL and co-existing medical conditions. Compared with patients treated with obinutuzumab plus chlorambucil, the obinutuzumab plus venetoclax combination was associated with a statistically significant improvement in progression-free survival (PFS; hazard ratio = 0.33; 95% CI 0.22-0.51; p<0.0001). After a median follow-up of 28 months (range not reported), median PFS was not reached in either arm.
The overall response rate also was higher in the venetoclax arm than the chlorambucil arm (85% vs. 71%; p=0.0007).
The most common AEs (≥20%) associated with venetoclax treatment were neutropenia, thrombocytopenia, anemia, diarrhea, nausea, upper respiratory tract infection, cough, musculoskeletal pain, fatigue, and edema.
Venetoclax was approved for this indication through the FDA’s new Real-Time Oncology Review pilot program.