The Food and Drug Administration (FDA) has accepted a supplemental new drug application (sNDA) for brukinsa (zanubrutinib; BeiGene, Ltd.) for the treatment of adults with Waldenström Macroglobulinemia (WM). Under the Prescription Drug User Fee Act (PDUFA), the agency is scheduled to issue a decision on the Bruton tyrosine kinase (BTK) inhibitor’s sNDA by October 18, 2021.1

The regulatory filing is based on safety and efficacy data from the phase 3 ASPEN trial (NCT03053440), in which investigators compared brukinsa monotherapy with single-agent ibrutinib in 351 patients with WM. The sNDA also includes data from a phase 2 trial (NCT03332173) of brukinsa in a population of Chinese patients with relapsed/refractory disease, a phase 1/2 evaluation in patients with B-cell malignancies (NCT02343120), and safety data from 779 patients across 6 clinical trials testing brukinsa.

The monotherapy is currently also under regulatory review for patients with WM in the European Union, Canada, Australia, China, Taiwan, and South Korea, according to BeiGene, Ltd., brukinsa’s developer.

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WM is a rare indolent B-cell lymphoma that presents in fewer than 2% of patients with non-Hodgkin lymphoma; an estimated 5000 new WM diagnoses are made each year in the United States.1 BTK inhibition has therapeutic activity in WM, and brukinsa, a second-generation BTK inhibitor, has been shown to be more selective than ibrutinib with fewer off-target effects.2

Brukinsa currently holds approval in the mantle cell lymphoma (MCL) space. On November 14, 2019, the FDA awarded brukinsa capsules an accelerated approval for the treatment of adults with MCL who received at least one prior therapy.3


  1. BeiGene announces U.S. FDA acceptance of supplemental new drug application for brukinsa (zanubrutinib) in Waldenström’s Macroglobulinemia. [press release]. Cambridge, MA and Beijing, CN: BeiGene, Ltd.; February 17, 2021.
  2. Zanubrutinib for the treatment of patients with Waldenström macroglobulinemia: 3 years of follow-up. Blood. 2020;136(18):2027-2037. doi: 10.1182/blood.2020006449
  3. FDA approves therapy to treat patients with relapsed and refractory mantle cell lymphoma supported by clinical trial results showing high response rate of tumor shrinkage. [press release]. Silver Spring, MD: FDA; November 14, 2019.