FDA Accepts BLA for bluebird and BMS' Multiple Myeloma CAR-T Treatment


Bristol Myers Squibb and bluebird bio are eying a potential quick approval for a CAR-T treatment for multiple myeloma after the U.S. Food and Drug Administration accepted the Biologics License Application under priority review.

For the two companies, the FDA’s decision is a turnaround from May when the regulatory agency declined to review the BLA for idecabtagene vicleucel (ide-cel; bb2121) first submitted in March. The FDA set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021.

Stanley Frankel, senior vice president of Cellular Therapy Development at BMS, said the priority review recognizes the potential of Ide-cel as the first anti-BCMA CAR T cell therapy to address a critical unmet need of patients with multiple myeloma.

Ide-cel is an 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. BCMA is a protein that is expressed on most multiple myeloma cancer cells, which makes it an important target for the treatment of this blood cancer. Ide-cel is the first CAR-T cell therapy submitted for regulatory approval to target this antigen and for multiple myeloma. BMS gained the drug through its $74 billion acquisition of Celgene.

The BLA is based on results from the pivotal Phase II 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. Topline data from the KarMMa study hit its primary endpoint of overall survival, as well as a secondary endpoint of complete response rate in this patient population. In its Refusal to File letter issued in March, the FDA said it needed additional data in order to review the BLA. The regulatory agency pointed to the Chemistry, Manufacturing and Control (CMC) module as the area that needs the additional data.

“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,” Joanne Smith-Farrell, chief operating officer oncology at bluebird bio said in a statement. “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.”

For those who have Celgene stock prior to the BMS acquisition, the FDA’s decision is crucial. FDA approval of ide-cel by March 31, 2021 is one of the required remaining milestones of the Contingent Value Rights issued upon the close of the Celgene acquisition. Another drug in the deal, liso-cel, has faced delays. Earlier this year, the FDA delayed review of the BLA for liso-cel by three months. The new PDUFA date is Nov. 16. Under CVR terms, liso-cel must be approved by Dec. 31.

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