The companies are seeking approval for the use of ide-cel in adult patients with the disease who have been subjected to least three prior therapies. The investigational cell therapy is intended to be used in patients who had undergone treatment with an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody.
The FDA is set to take a decision on the cell therapy candidate by 27 March 2021, which is its Prescription Drug User Fee Act (PDUFA) goal date.
Ide-cel is a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, which has a breakthrough therapy designation (BTD) by the FDA for relapsed and refractory multiple myeloma.
In the European Union, the cell therapy candidate has PRIority MEdicines (PRIME) designation for the same indications.
Bristol Myers Squibb cellular therapy development senior vice president Stanley Frankel said: “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.
“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 biologics license application, which has been accepted for priority review by the FDA, is based on the findings of the phase 2 KarMMa study.
The mid-stage trial assessed the efficacy and safety of ide-cel in 128 adults with heavily pre-treated and highly refractory multiple myeloma. The enrolled patients were all previously exposed to an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody.
bluebird bio oncology chief operating officer Smith-Farrell said: “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.”