The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for idecabtagene vicleucel (ide-cel [bb2121]; Bristol Myers Squibb and bluebird bio, Inc) for the treatment of adults with multiple myeloma who have received at least 3 prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.
Ide-cel is an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy that works by binding to BCMA on the surface of multiple myeloma cells resulting in CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.
The BLA is supported by data from the open-label, single-arm phase 2 KarMMa study that evaluated the efficacy and safety of ide-cel in 128 adults with relapsed and refractory multiple myeloma who had received at least 3 prior therapies. Patients received a target dose range of 150-450 x 106 CAR+ T cells after lymphodepleting chemotherapy. The primary end point was the overall response rate (ORR) as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria.
Results showed that at a median follow up of 11.3 months, the ORR was 73% and median progression-free survival (PFS) was 8.6 months; ORR and PFS increased with higher doses of ide-cel. In subgroup analyses, which included older and high-risk patients, the ORR was observed to be at least 50%. As for safety, the most common toxicities of any grade were cytopenias (97%) and cytokine release syndrome (84%).
“Today’s Priority Review milestone recognizes the potential of this first anti-BCMA CAR T cell therapy to address a critical unmet need of patients with multiple myeloma,” said Stanley Frankel, MD, senior vice president, Cellular Therapy Development, Bristol Myers Squibb. “We are pleased by the significant progress that is being made in partnership with patients and the multiple myeloma community to bring ide-cel to adults with relapsed and refractory multiple myeloma who are triple-class exposed and may benefit from an important new therapeutic option.”
A Prescription Drug User Fee Act (PDUFA) target date of March 27, 2021 has been assigned for this application.
US Food and Drug Administration (FDA) accepts for Priority Review Bristol Myers Squibb and bluebird bio application for anti-BCMA CAR T cell therapy idecabtagene vicleucel (Ide-cel, bb2121). https://www.businesswire.com/news/home/20200922005376/en/U.S.-Food-and-Drug-Administration-FDA-Accepts-for-Priority-Review-Bristol-Myers-Squibb-and-bluebird-bio-Application-for-Anti-BCMA-CAR-T-Cell-Therapy-Idecabtagene-Vicleucel-Ide-cel-bb2121. Accessed September 22, 2020.