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)
Bristol Myers Squibb (NYSE: BMY) and bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review their Biologics License Application (BLA) for idecabtagene vicleucel (ide-cel; bb2121), the companies’ investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021.
“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, M.D., 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.”
The BLA is based on results from the pivotal Phase 2 KarMMa study evaluating the efficacy and safety of ide-cel in 128 adults with heavily pre-treated and highly refractory multiple myeloma exposed to an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. Results from the study were shared during an oral presentation as part of the American Society of Clinical Oncology 2020 (ASCO20) Virtual Scientific Program.1
“Today’s acceptance of the BLA for ide-cel for Priority Review by the FDA marks a key moment in our journey to bring this BCMA-directed CAR T cell therapy to multiple myeloma patients who are in desperate need of new options,” said Joanne Smith-Farrell, Ph.D., chief operating officer oncology, bluebird bio. “Based on the body of evidence we have generated in an advanced, heavily pre-treated patient population, our confidence in the potential of ide-cel as an important treatment option remains high. Together with our partners at Bristol Myers Squibb, we are committed to continue working with the FDA to deliver this promising therapy to patients in an expeditious manner.”