BeOne Medicines announced that the FDA has granted accelerated approval to BEQALZI (sonrotoclax), a 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. The decision makes BEQALZI the first and only BCL2 inhibitor approved specifically for R/R MCL in the United States and the first new BCL2 inhibitor to reach the U.S. market in a decade.
BEQALZI was rationally designed to enhance BCL2 inhibition, with greater potency and selectivity and a pharmacologic profile intended to improve efficacy, tolerability, and convenience relative to earlier agents in the class. The FDA’s accelerated approval is based on results from the Phase 1/2 BGB‑11417‑201 trial (NCT05471843), which evaluated sonrotoclax monotherapy in adults with R/R MCL previously treated with a BTK inhibitor.
In the study, an independent review showed an overall response rate (ORR) of 52% (95% CI, 42–62), including a complete response (CR) rate of 16% (95% CI, 9.1–24.0). Median time to response was 1.9 months, and median duration of response was 15.8 months (95% CI, 7.4 months–not estimable) at a median response follow‑up of 11.9 months, with follow‑up still maturing. Sonrotoclax monotherapy was described as generally well tolerated in the trial.
The FDA previously granted sonrotoclax Breakthrough Therapy, Fast Track, and Orphan Drug designations for R/R MCL, recognizing the high unmet need in patients who have progressed after BTK inhibition, where outcomes are typically poor and treatment options are limited. The new approval is contingent on verification of clinical benefit in the ongoing, confirmatory Phase 3 CELESTIAL‑RRMCL trial (NCT06742996).
BEQALZI is already approved in China for the treatment of R/R MCL and for adults with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) who have received at least one prior systemic therapy, including a BTK inhibitor, and filings based on the Phase 1/2 data are under review by the European Medicines Agency and other global regulators. BeOne describes BEQALZI as a foundational, potentially best‑in‑class BCL2 inhibitor with a unique pharmacokinetic and pharmacodynamic profile, including high potency and specificity, a short half‑life, and no drug accumulation in early clinical studies.
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