FDA Accepts Anti-BCMA Ide-cel for MM for Priority Review

September 22, 2020

FDA will review the first chimeric antigen receptor (CAR) T cell immunotherapy for the treatment of multiple myeloma (MM) in adults who have not responded or have relapsed after at least 3 other therapies.

Bristol Myers Squibb and bluebird bio said Tuesday the FDA accepted and granted priority review for the Biologics License Application (BLA) for idecabtagene vicleucel (ide-cel; bb2121), the companies’ B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy.

If approved, it would be first CAR T cell immunotherapy for the treatment of multiple myeloma (MM) in adult patients who have not responded or have relapsed after at least 3 other targeted therapies.

The Prescription Drug User Fee Act (PDUFA) date is 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,” Stanley Frankel, MD, senior vice president, Cellular Therapy Development, Bristol Myers Squibb, said in a statement. “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.”

These patients have previously received at least 3 prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody.

Ide-cel functions by recognizing and binding to BCMA on the surface of MM cells. This leads to CAR T-cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

“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,” added Joanne Smith-Farrell, PhD, 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.”

The application is based on data from the pivotal phase 2 KarMMa study, which included a total of 140 patients, 128 of whom received ide-cel across target dose levels of 150-450 x 106 CAR+ T cells. Eighty-four percent of patients enrolled were refractory to all 3 classes of treatments and 94% were refractory to anti-CD38 antibodies specifically.

Overall response rate was 73% across all dose levels, including 33% of patients who had a complete response (CR) or stringent CR (sCR); nearly all subgroups had an ORR of 50% or greater, including older and high-risk patients

The median duration of response (DoR) was 10.7 months, with a 19-month median DoR for patients who had a CR or sCR, and median progression-free survival (PFS) was 8.8 months, with a 20.2-month median PFS for patients who had a CR or sCR.

Ide-cel was previously granted breakthrough therapy designation by the FDA, and PRIority MEdicines designation and validation of its Marketing Authorization Application by the European Medicines Agency. Bristol Myers Squibb plans regulatory submissions for ide-cel in additional markets outside the United States and European Union.