BMS and bluebird bio Submit BLA for Ide-cel, the First Anti-BCMA CAR-T Therapy, to FDA

On April 1, Bristol-Myers Squibb (BMS) and bluebird bio jointly announced that they had submitted a Biologics License Application (BLA) to the U.S. FDA for their CAR-T therapy idecabtagene vicleucel (ide-cel, also known as bb2121) for the treatment of patients with relapsed/refractory multiple myeloma (R/R MM). The press release noted that ide-cel is the first BCMA-targeted CAR-T therapy to submit a marketing application.

Multiple myeloma (MM) is a malignant plasma cell disorder characterized by abnormal proliferation of plasma cells in the bone marrow and excessive production of monoclonal immunoglobulins. These abnormal plasma cells accumulate in the bone marrow and can form tumors in multiple bone sites throughout the body. Not only do these cells fail to perform normal functions, but the antibodies they produce also impair the bone marrow’s ability to generate healthy blood cells. In addition, patients often present with multiple osteolytic lesions, hypercalcemia, anemia, and renal impairment. Although various therapies are currently available for multiple myeloma, many patients still develop resistance to all approved treatments, underscoring the critical importance of developing innovative therapeutic approaches for this disease.

B-cell maturation antigen (BCMA) is a protein widely expressed on the cancer cells of multiple myeloma, making it an important potential target for this aggressive blood cancer. Ide-cel can recognize and bind to BCMA on the surface of multiple myeloma cells, leading to the proliferation and differentiation of CAR-T cells, which subsequently kill cells expressing BCMA. Previously, ide-cel was granted Breakthrough Therapy designation by the FDA and received PRIME designation from the European Medicines Agency.

▲Key efficacy data for Ide-cel (Image source: Reference [2])

The submission of this marketing application is based on the positive results from the pivotal, single-arm, open-label Phase 2 KarMMa clinical trial. Patients with relapsed/refractory multiple myeloma (R/R MM) enrolled in the trial had previously received at least immunomodulatory drugs (IMiDs), proteasome inhibitors (PIs), and anti-CD38 antibodies, and had developed resistance to these therapies. Among them, 94% of patients were refractory to anti-CD38 antibody therapy, and 84% exhibited triple-class refractoriness. The trial results demonstrated that among a total of 128 efficacy-evaluable patients, the overall response rate (ORR) was 73.4%, and the complete response rate (CR) was 31.3%. With a median follow-up duration of 11.3 months, the progression-free survival (PFS) for these patients was 8.6 months.

We look forward to the successful launch of ide-cel, bringing benefit to a broad population of patients in need!

References:

[1] Bristol Myers Squibb and bluebird bio Announce Submission of Biologics License Application (BLA) for Anti-BCMA CAR T Cell Therapy Idecabtagene Vicleucel (Ide-cel, bb2121) to FDA, Retrieved March 31, 2020, from https://www.businesswire.com/news/home/20200331005194/en

[2] Bristol-Myers Squibb and bluebird bio Announce Positive Top-line Results from the Pivotal Phase 2 KarMMa Study of Ide-cel in Relapsed and Refractory Multiple Myeloma. Retrieved March 31, 2020, from https://www.businesswire.com/news/home/20191206005463/en/Bristol-Myers-Squibb-bluebird-bio-Announce-Positive-Top-line

Original Title: Express | The First BCMA-Targeting CAR-T Therapy Is Coming, BMS Submits Marketing Application

▽Follow [WuXi AppTecDe】WeChat Official Account