BeiGene, Ltd., a commercial-stage biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that the U.S. Food and Drug Administration (FDA) has accepted a supplemental new drug application (sNDA) for BRUKINSA (zanubrutinib) for the treatment of adult patients with Waldenström’s Macroglobulinemia (WM). The Prescription Drug User Fee Act (PDUFA) target action date is October 18, 2021.

“We are pleased that the FDA has accepted the sNDA for BRUKINSA in WM, a rare disease with significant morbidity. BTK inhibitors have transformed the treatment of WM in recent years, but discrepancies in response exist in patients with different subtypes, and toxicity can remain an issue,” said Jane Huang, M.D., Chief Medical Officer, Hematology, at BeiGene. “We look forward to continuing our communications with the FDA in the coming months and hope that BRUKINSA will become a new treatment option for patients with WM in the United States.”

The sNDA package, which includes data from 351 patients with WM, was primarily based on safety and efficacy data from the global Phase 3 ASPEN trial of zanubrutinib compared to ibrutinib for the treatment of WM (NCT03053440), with supportive data from the pivotal Phase 2 trial of zanubrutinib in relapsed/refractory WM conducted in China (NCT03332173) and the global Phase 1/2 trial in patients with B-cell malignancies (NCT02343120). In addition, safety data from 779 patients in six clinical trials of BRUKINSA were included in the submission.

In addition to the United States, BRUKINSA is also under regulatory review as a treatment for patients with WM in the European Union, Canada, Australia, China, Taiwan, and South Korea.

In November 2019, BRUKINSA received accelerated approval in the United States as a treatment for mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy. In June 2020, BRUKINSA received conditional approval in China as a treatment for adult patients with chronic lymphocytic leukemia (CLL) /small lymphocytic lymphoma (SLL) who have received at least one prior therapy, and as a treatment for adult patients with MCL who have received at least one prior therapy. Currently, more than 20 marketing applications for BRUKINSA have been submitted, covering 45 countries and regions globally, including the United States, China, and European Union.

About Waldenström’s Macroglobulinemia

Waldenström’s macroglobulinemia (WM) is a rare indolent B-cell lymphoma that occurs in less than two percent of patients with non-Hodgkin’s lymphoma (NHL). There are about 5,000 new cases of WM diagnosed each year in the United States. The disease usually affects older adults and is primarily found in the bone marrow, although lymph nodes and the spleen may be involved.i

About BRUKINSA (zanubrutinib)

BRUKINSA (zanubrutinib) is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK), discovered by BeiGene scientists, that is currently being evaluated globally in a broad pivotal clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies.

BRUKINSA was approved in the United States in November 2019 to treat adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. A supplemental new drug application (sNDA) of BRUKINSA in patients with Waldenström’s macroglobulinemia (WM) has been accepted for review by the FDA.

BRUKINSA received conditional approval in China in June 2020 for the treatment of MCL in adult patients who have received at least one prior therapy and the treatment of CLL/SLL in adult patients who have received at least one prior therapy. Complete approval for these indications may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials. An sNDA of BRUKINSA in patients with relapsed/refractory WM has been accepted by the Center for Drug Evaluation (CDE) of the National Medical Products Administration and is currently under priority review.

A marketing authorization application (MAA) for BRUKINSA for the treatment of patients with WM who have received at least one prior therapy or as first-line treatment for patients unsuitable for chemo-immunotherapy has been accepted for review by the European Medicines Agency (EMA). In addition, 20 marketing applications for BRUKINSA have been submitted in 16 countries and regions.