FDA Grants Breakthrough Designation to Mantle Cell Lymphoma Treatment

Zanubrutinib (BGB-3111) is being investigated for the treatment of various B-cell malignancies, including mantle cell lymphoma.

BeiGene’s investigational Bruton’s tyrosine kinase (BTK) inhibitor, zanubrutinib (BGB-3111), has been granted Breakthrough Therapy designation by the FDA for the treatment of adults with mantle cell lymphoma (MCL) who have received at least 1 prior therapy, according to a company press release.1

MCL is an aggressive form of non-Hodgkin lymphoma (NHL) that arises from B-cells originating in the “mantle zone,” according to BeiGene. Approximately 70,800 new cases of NHL were estimated in the United States in 2014, with MCL accounting for approximately 6%.1

The designation is based on data presented at the 60th Annual Meeting of the American Society of Hematology, which took place December 1-4, 2018. Results from the phase 2 trial of zanubrutinib in Chinese patients with relapsed or refractory MCL demonstrated a high rate of complete responses characterized by positron emission tomography-based imaging. For the study, patients were treated with zanubrutinib, dosed at 160 mg orally twice-daily.

Key findings include2:

  • The overall response rate was 83.5%, the complete response rate was 58.8%, and the partial response rate was 24.7% for zanubrutinib.
  • The 24-week progression-free survival was estimated at 82%.
  • With 24.1 weeks median follow-up, the median duration of response had not yet been reached and 90% of responders were still in response at 24 weeks.

Additionally, zanubrutinib demonstrated tolerability that was consistent with previous reports in patients with B-cell malignancies. The most common adverse events reported were decreased neutrophil count (31.4%), rash (29.1%), upper respiratory tract infection (29.1%), and decreased platelet count (22.1%).2

In a global phase 1 trial, zanubrutinib was evaluated as a monotherapy in patients with different subtypes of B-cell malignancies, including MCL. The data showed consistent outcomes for patients outside of China.2

Clinical trials are also investigating the use of zanubrutinib in Waldenström macroglobulinemia (WM), previously untreated chronic lymphocytic leukemia, small lymphocytic leukemia, and relapsed/refractory follicular lymphoma. Zanubrutinib has previously been granted Fast Track designation for the treatment of patients with WM.2

“We are very excited to receive the Breakthrough Therapy designation from the FDA,” Jane Huang, MD, chief medical officer of hematology at BeiGene, said in a statement.1 “Zanubrutinib has been designed to maximize BTK occupancy and minimize off-target effects. We believe that the Breakthrough Therapy designation underscores the potential of zanubrutinib as a meaningful treatment for patients with MCL who have received at least 1 prior therapy.”


  • BeiGene Receives U.S. FDA Breakthrough Therapy Designation for Zanubrutinib in Mantle Cell Lymphoma [news release]. BeiGene’s website. http://ir.beigene.com/phoenix.zhtml?c=254246&p=irol-newsArticle&ID=2383285. Accessed January 15, 2019.
  • BeiGene Announces Clinical Results of Zanubrutinib in Mantle Cell Lymphoma From Two Presentations at the 60th American Society of Hematology Annual Meeting [news release]. BeiGene’s website. http://ir.beigene.com/phoenix.zhtml?c=254246&p=irol-newsArticle&ID=2378923. Accessed January 15, 2019.

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