The U.S. Food and Drug Administration (FDA) granted approval to venetoclax for the treatment of patients with previously treated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), regardless of del17p status. The BCL2 inhibitor was previously approved in April 2016 for the treatment of patients with previously treated CLL/SLL who also harbored a del17p mutation.
The agency’s decision was based on data from the phase III MURANO randomized trial that compared venetoclax with bendamustine, in combination with rituximab, in 389 patients. In the venetoclax group, the response rate was 92 percent, compared with 72 percent for those treated with bendamustine. At a median follow-up of 23 months (range not reported), progression-free survival was not reached in the venetoclax group, compared with 18.1 months in the bendamustine group (hazard ratio = 0.19; 95% CI 0.13-0.28; p<0.0001).
In patients receiving the venetoclax combination, the most common adverse events (AEs) included neutropenia, diarrhea, upper respiratory–tract infection, fatigue, cough, and nausea. Serious AEs occurred in 46 percent of patients, and 21 percent of patients developed serious infections. The investigators also noted that rapid reduction in tumor volume makes tumor lysis syndrome an important risk to consider.
Source: FDA press release, June 8, 2018.