The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to bb2121 — a chimeric antigen receptor T-cell (CAR-T) therapy that targets B-cell maturation antigen (BCMA) — for patients with previously treated multiple myeloma (MM), according to a news release from Celgene Corporation.

The FDA based its approval on preliminary evidence from the ongoing CRB-401 phase 1 study. For this study, researchers infused 21 patients with MM who had received at least 3 previous lines of therapy with bb2121.

After a median follow-up of 15.4 weeks, the overall response rate was 89%, and increased to 100% for patients treated with 150×106 CAR+ T-cells or greater. Patients who were treated with 150×106 CAR+ T-cells or higher did not experience disease progression with time between 8 and 54 weeks since bb2121 exposure.

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At the time of data cut-off, no grade 3 or higher treatment-emergent neurotoxicites were reported. Primarily grade 1 to 2 cytokine release syndrome (CRS) was observed in 71% of patients; 2 patients experienced grade 3 CRS that resolved in 24 hours; and 4 patients were treated with tocilizumab, and 1 with steroids, to manage their CRS symptoms.

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Breakthrough Therapy Designation is granted by the FDA to agents that may significantly improve at least one clinical outcome for a serious or life-threatening disease in order to quicken the development, review, and potential approval of these medications.


1. Celgene Corporation and bluebird bio announce bb2121 anti-BCMA CAR-T cell therapy has been granted breakthrough therapy designation from FDA and prime eligibility from EMA for relapsed and refractory multiple myeloma [news release]. Summit, NJ; Cambridge, MA: Celgene Corporation; bluebird bio; November 16, 2017. Accessed November 27, 2017.