Ide-cel is an investigational anti-BCMA CAR T cell immunotherapy, developed under partnership between Bristol Myers Squibb and bluebird bio
Entrance to the Bristol-Myers Squibb building. (Credit: Jonathan Schilling/Wikipedia.)
Bristol Myers Squibb and bluebird bio have secured the US Food and Drug Administration (FDA) Priority Review for idecabtagene vicleucel (Ide-cel) to treat a type of multiple myeloma in adults.
Ide-cel is an investigational B-cell maturation antigen targeting (anti-BCMA) chimeric antigen receptor (CAR) T cell immunotherapy, developed under a co-development, co-promotion and profit share partnership between Bristol Myers Squibb and bluebird bio.
The US regulatory agency has accepted the companies’ Biologics License Application (BLA) for Ide-cel to treat multiple myeloma in adults who received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.
The FDA has selected 27 March 2021 as Prescription Drug User Fee Act (PDUFA) goal date.
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 Ide-cel BLA is based on results from the Phase 2 KarMMa clinical trial
The filing of BLA is based on results from the KarMMa, an open-label, single-arm, multicentre, Phase 2 study evaluating the efficacy and safety of ide-cel in 128 adults with relapsed and refractory multiple myeloma in North America and Europe
Overall response rate (ORR), as measured by an independent review committee (IRC), according to the International Myeloma Working Group (IMWG) criteria is the primary endpoint complete response rate is an important secondary endpoint of the clinical trial.
Time and duration of response, progression-free survival, overall survival, minimal residual disease, measured by an NGS assay and safety include other secondary endpoints.
Ide-cel has received breakthrough therapy designation (BTD) from the FDA and priority medicines (PRIME) designation and got its marketing authorisation application (MAA) validated by the European Medicines Agency (EMA) for relapsed and refractory multiple myeloma indication.
Bristol Myers Squibb said that its Ide-cel has not been approved for any indication in any country, and is planning to submit for regulatory approval in markets outside the US and EU.
bluebird bio chief operating officer oncology Joanne Smith-Farrell said: “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.
“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.”