FDA approves Brukinsa for treatment of Waldenström’s Macroglobulinemia.- BeiGene Ltd

“The ASPEN trial provided compelling evidence that Brukinsa is a highly active BTK inhibitor in Waldenström’s macroglobulinemia, and compared to the first-generation BTK inhibitor, showed improved tolerability across a number of clinically important side effects. The approval of Brukinsa provides an important new option for targeted therapy in Waldenström’s macroglobulinemia,” said Steven Treon, M.D., Ph.D., Director of the Bing Center for Waldenström’s Macroglobulinemia Research at the Dana-Farber Cancer Institute and Professor of Medicine at Harvard Medical School.

The FDA’s approval of Brukinsa in WM is primarily based on efficacy results from the multicenter, open-label Phase III ASPEN trial (NCT03053440) comparing Brukinsa to ibrutinib in patients with WM. The primary efficacy endpoint of the ASPEN trial was very good partial response (VGPR) rate in the overall intention-to-treat (ITT) population as assessed by independent review committee (IRC). Based on the modified Sixth International Workshop on Waldenström’s Macroglobulinemia (IWWM-6) response criteria (Treon 2015), the VGPR rate was 28% with Brukinsa, compared to 19% with ibrutinib; based on the IWWM-6 response criteria (Owen et al 2013), the VGPR rate was 16% with Brukinsa, compared to 7% with ibrutinib.