Benzinga | Mar 2, 2021 07:16AM EST

Health Canada Approves BRUKINSA for the Treatment of Waldenstrm's Macroglobulinemia

* Approval is based on the Phase 3 ASPEN trial of BRUKINSA compared to ibrutinib

* This marks the first regulatory approval for BRUKINSA in Waldenstr?m's macroglobulinemia, and BeiGene's first approval in Canada

BeiGene, Ltd. (NASDAQ:BGNE, HKEX: 06160))), a commercial-stage biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that BRUKINSA(r) (zanubrutinib) has been approved by Health Canada for the treatment of adult patients with Waldenstr?m's macroglobulinemia (WM).

"BRUKINSA is a highly selective BTK inhibitor designed to provide deep and durable response for patients with hematologic malignancies while reducing the frequency of certain side effects. With today's approval, we are looking forward to bringing this potentially best-in-class BTK inhibitor to Canadians affected by WM," said Josh Neiman, Chief Commercial Officer for North America and Europe at BeiGene. "We are committed to working to ensure access for patients in Canada and to making BRUKINSA available to patients in more markets worldwide."

"WM is a rare disease with significant morbidity. BTK inhibitors have brought advancements in the treatment of WM, yet not all patients respond and intolerability due to side effects remains an issue, particularly for the elderly patient population," said Christine Chen, M.D., Med, FRCPC, Associate Professor at University of Toronto and Clinical Investigator at Princess Margaret Cancer Centre. "The ASPEN trial results underscore the potential that zanubrutinib has to provide clinical benefit with advantages in safety, offering new hope for WM patients."

"The Waldenstr?m's Macroglobulinemia Foundation of Canada (WMFC) is delighted with Health Canada's approval of BRUKINSA (zanubrutinib) as a WM treatment. This marks an important step forward in providing a variety of quality options for Canadian patients. As the study results from ASPEN demonstrated, BRUKINSA presents the possibility of improved outcomes for Canadian patients," commented Paul Kitchen, Chair of Board at WMFC.

Following the previously granted priority review in September 2020, the Health Canada approval for BRUKINSA is based on efficacy results from the ASPEN clinical trial, a Phase 3 randomized, open-label, multicenter trial (NCT03053440) that evaluated BRUKINSA compared to ibrutinib in patients with relapsed/refractory (R/R) or treatment-na?ve (TN) WM who harbor a MYD88 mutation (MYD88MUT). In the ASPEN trial, BRUKINSA demonstrated numerically higher very good partial response (VGPR) rate and a favorable safety profile over ibrutinib, although the primary endpoint of statistical superiority related to deep response (VGPR or better) was not met.

As assessed by independent review committee (IRC) per adaptation of the response criteria updated at the Sixth International Workshop on Waldenstr?m's Macroglobulinemia (IWWM), the combined complete response (CR) + VGPR rate in the overall intention-to-treat (ITT) population was 28.4% with BRUKINSA (95% CI: 20, 38), compared to 19.2% with ibrutinib (95% CI: 12, 28).1

In the ASPEN trial, of the 101 patients with WM randomized and treated with BRUKINSA, four percent of patients discontinued due to adverse events, including cardiomegaly, neutropenia, plasma cell myeloma, and subdural hemorrhage. Adverse events leading to dose reduction occurred in 14% of patients, with the most common being neutropenia (3.0%) and diarrhea (2.0%).1

The overall safety profile of BRUKINSA is based on pooled data from 779 patients with B-cell malignancies treated with BRUKINSA in clinical trials. The most common adverse reactions (?10%) with BRUKINSA were neutropenia, thrombocytopenia, upper respiratory tract infection, anemia, rash, musculoskeletal pain, diarrhea, cough, contusion, pneumonia (grouped terms), urinary tract infection, hemorrhage (grouped terms), and hematuria. The most frequent serious adverse reactions (?2%) were pneumonia (10.0%) and hemorrhage (2.1%).1

The recommended total daily dose of BRUKINSA is 320mg. BRUKINSA is expected to be available in Canada in the coming weeks.