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BeOne Medicines Ltd, a global oncology company, announced that the US Food and Drug Administration (FDA) has granted accelerated approval to Beqalzi (bee-KAHL-zee; sonrotoclax), a foundational, next-generation BCL2 inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL), after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor. Beqalzi was designed to enhance BCL2 inhibition - with greater potency, selectivity, and a pharmacologic profile with potential to improve efficacy, tolerability, and convenience over others in the class.
Michael Wang, MD, Global Principal Investigator, the Puddin Clarke Endowed Professor, Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, said: “The data supporting the approval of sonrotoclax in the US confirm its role as a foundational therapy for mantle cell lymphoma in the post-BTK inhibitor setting, and demonstrate that it can deliver robust disease control when treatment choices are limited and outcomes are poor. From a clinical perspective, this provides physicians with an important new option grounded in both efficacy and tolerability, fundamentally changing how we think about sequencing therapy in this disease.”
The accelerated approval of Beqalzi is supported by efficacy and safety data from the phase 1/2 study, BGB-11417-201 (NCT05471843), which was presented at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition. The study included an independent review of efficacy data and demonstrated:
- Overall response rate (ORR): 52% (95% CI, 42-62).
- Complete response (CR) rate: 16% (95% CI, 9.1-24.0).
- Median time to response (TTR): 1.9 months.
- Median duration of response (DOR): 15.8 months (95% CI, 7.4 months-NE) at a median response follow-up of 11.9 months (has yet to reach full maturity)
- Safety: treatment with sonrotoclax monotherapy was generally well tolerated.
Continued approval for this indication is contingent upon confirmation of clinical benefit in the confirmatory CELESTIAL-RRMCL trial (NCT06742996), which is underway. The US FDA granted Breakthrough Therapy Designation (BTD) for sonrotoclax in this indication, as well as Fast Track Designation and Orphan Drug Designation.
Amit Agarwal, CMO, Hematology, BeOne Medicines, said: “BeOne is leading the advancement and enhancement of BCL2 inhibition to revolutionize how we treat patients living with B-cell malignancies. Today’s approval of Beqalzi represents critical progress for patients with mantle cell lymphoma and reinforces our strategy of building foundational medicines designed to raise the standard of care in B-cell malignancies.”
MCL is a rare and often aggressive subtype of non-Hodgkin lymphoma. In the United States, approximately 3,300 new cases of MCL are diagnosed each year. While many patients respond to initial therapy, relapse is common, and outcomes after progression can be poor, particularly after prior treatment with a BTK inhibitor. The accelerated approval of Beqalzi introduces a new targeted mechanism to the MCL treatment landscape and reinforces the importance of expanding therapeutic choices for patients as the disease evolves.
Meghan Gutierrez, CEO, Lymphoma Research Foundation, said: “For people living with relapsed or refractory mantle cell lymphoma, each progression can bring uncertainty and questions regarding remaining treatment options. The FDA approval of sonrotoclax represents significant progress for the US mantle cell lymphoma community, offering renewed hope for patients and families who have exhausted other available therapies. Advances like this underscore why continued research and innovation in this disease remain so critical.”
Beqalzi is also approved in China for the treatment of R/R MCL, as well as adult patients with chronic lymphocytic leukaemia (CLL)/small lymphocytic lymphoma (SLL) who have previously received at least one systemic therapy, including a BTK inhibitor. Data from the phase 1/2 study of sonrotoclax in R/R MCL is also under review by the European Medicines Agency and other regulatory agencies.
The US FDA also granted sonrotoclax Fast Track Designation for Waldenström macroglobulinemia (WM), as well as Orphan Drug Designation for the treatment of adult patients with WM, multiple myeloma, acute myeloid leukaemia, and myelodysplastic syndrome.
Additionally, sonrotoclax is currently being studied in combination with other therapeutics, including zanubrutinib, as a potential treatment for CLL, with updated data expected to be presented at the 2026 American Society of Clinical Oncology Annual Meeting.
Beqalzi (sonrotoclax) is a foundational, next-generation and potentially best-in-class B-cell lymphoma 2 (BCL2) inhibitor with a unique pharmacokinetic and pharmacodynamic profile. Preclinical and clinical studies in early drug development have shown that sonrotoclax is a highly potent and specific BCL2 inhibitor with a short half-life and no drug accumulation. Sonrotoclax has shown promising clinical activity across a range of B-cell malignancies, including chronic lymphocytic leukaemia (CLL), and is in development as a monotherapy and in combination with other therapeutics, including zanubrutinib. To date, more than 2,200 patients have been enrolled across the broad sonrotoclax global development programme.
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