Second-Ever Therapy for Waldenström’s Macroglobulinemia is Now FDA Approved

The FDA has just announced their approval of zanubrutinib (Brukinsa) as a therapeutic option for Waldenström’s macroglobulinemia. This approval was given based on the ASPEN Phase 3 clinical trial, and it is only the second approved therapy for this condition.


This trial (NCT03053440) was a multi center and open-label trial which enrolled 201 patients diagnosed with Waldenström’s macroglobulinemia who had a MYD88-mutation. Approximately 90% of patients with this rare disease have this mutation.

This investigation compared the new investigative therapy zanubrutinib with ibrutinib. The primary endpoint was very good partial response (VGPR).

Results were extremely promising, demonstrating that Brukinsa is a very active BTK inhibitor and greatly improves patient outcomes. The therapy was also found to be well tolerated, and patients in the trial had minimal side effects. Common AEs were low platelet count, hemorrhage, diarrhea, cough, pneumonia, low hemoglobin, musculoskeletal pain, bruising, low neutrophil count, and respiratory infection.

ASPEN demonstrated that treatment with zanubrutinib (Brukinsa) produced a high VGPR. Specifically, VGPR was 28% compared to 19% for those treated with ibrutinib. When utilizing the IWWM criteria to compare, zanubrutinib had a VGPR of 16% and ibrutinib had a VGPR of 7%.

Also using this criteria, zanubrutinib had a response rate of 78% and ibrutinib also had a response rate of 78%. The patients treated with zanubrutinib had an event-free duration of response in a one year period of 94% compared to 88% in the ibrutinib group.

Looking Forward

Researchers and patients are excited about this new therapeutic option. Not only are they excited by this therapy’s efficacy, but they are also excited that it is an oral treatment. Oral therapies, which are a single agent, can help to greatly improve patient’s quality of life while managing their condition.

You can read more about this new approval here!


  1. U.S. FDA grants BRUKINSA® (Zanubrutinib) approval in Waldenström’s macroglobulinemia. News release. BeiGene, Ltd. September 1, 2021. Accessed September 1, 2021.
  2. Tam CS, Opat S, D’Sa S, et al. A randomized phase 3 trial of zanubrutinib vs ibrutinib in symptomatic Waldenström macroglobulinemia: the ASPEN study. Blood. 2020;136(18):2038–2050. doi:10.1182/blood.2020006844

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