This morning, the companies announced the regulatory agency issued a Refusal to File letter regarding the BLA for idecabtagene vicleucel (ide-cel; bb2121) for patients with heavily pretreated relapsed and refractory multiple myeloma.
Shares of bluebird bio are down nearly 7% in premarket trading after the U.S. Food and Drug Administration (FDA) declined to review a Biologics License Application for a CAR-T treatment for multiple myeloma co-developed with Bristol Myers Squibb.
This morning, the companies announced the regulatory agency issued a Refusal to File letter regarding the BLA for idecabtagene vicleucel (ide-cel; bb2121) for patients with heavily pretreated relapsed and refractory multiple myeloma. The two companies submitted the BLA in March.
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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.
In its Refusal to File letter, the FDA said it will need 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. The companies noted the FDA did not require any additional clinical or non-clinical data. Bristol Myers Squibb is planning to resubmit the BLA no later than the end of July 2020, the companies said in their brief announcement.
The delay for ide-cel is a bit worrisome for Celgene shareholders as the drug, along with two others, were timeline-specific for a contingent value right (CVR) payment of $9 a share if the drugs were approved by a particular date, Reuters noted. Ide-cel was supposed to be approved by March 31 to meet the terms of the CVR. Ide-cel isn’t the only drug that could derail the CVR. Another drug in the deal, liso-cel, is also facing a delay. Earlier this month, 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.
The BLA was based on results from the pivotal Phase II KarMMa study that assessed the efficacy and safety of ide-cel in heavily pre-treated patients with relapsed and refractory multiple myeloma. 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. Full results of the trial are expected to be presented at the virtual American Society of Clinical Oncology meeting later this month.
The Refusal to File letter came two days after bluebird announced an amended collaboration with BMS on ide-cel. Under terms of the deal, BMS paid bluebird $200 million for rights to royalties off of sales outside the United States. BMS will eventually assume manufacturing responsibilities for ide-cel outside of the United States, while bluebird will produce lentiviral vector to support the U.S. commercial market for ide-cel.
