BMS, bluebird refile FDA approval application for CAR-T therapy in myeloma

BMS, bluebird refile FDA approval application for CAR-T therapy in myeloma

Two companies that developed a CAR-T cell therapy for multiple myeloma have resubmitted their approval application to the Food and Drug Administration after the agency initially declined to file it two months ago.
Cambridge, Massachusetts-based bluebird bio and New York-based Bristol-Myers Squibb said in an after-hours announcement Wednesday that they had sent their application for idecabtagene vicleucel to the FDA. The CAR-T targets BCMA, a cell-surface protein widely expressed in multiple myeloma, and the companies are seeking approval of the cell therapy in patients who have failed treatment on an immunomodulatory agent, a proteasome inhibitor and a CD38-targeting monoclonal antibody, which are considered the “backbone” treatments of myeloma.
Shares of bluebird rose 2.6% in after-hours trading on the Nasdaq following the news Wednesday. BMS’ shares also rose slightly on the New York Stock Exchange.
The companies had sought approval of the drug earlier this year, but they hit a roadblock in mid-May when the FDA delivered a refuse-to-file letter requesting supplemental documentation related to chemistry, manufacturing and controls. However, it did not request new clinical or preclinical data. The companies said at the time that they intended to resubmit their application by the end of July.
In a note to investors Wednesday, RBC Capital Markets analyst Luca Issi wrote that bluebird had confirmed that it requested a priority review from the FDA for the application, noting that the FDA will decide within 60 days on whether to accept the application and whether to grant the priority review.
In Issi’s view, the FDA is likely to grant the priority review given the strength of data from the Phase II KarMMa study on which the application was based, which included a median progression-free survival of 12.1 months, compared with three months for the standard of care, along with its existing breakthrough therapy designation and an unmet need in late-line multiple myeloma. A priority review, he wrote, would result in an FDA decision as early as the first quarter of 2021, and likely in March.
Issi further wrote that bluebird and BMS have a first-mover advantage in a market worth $20 billion, with Johnson & Johnson and Legend Biotech’s BCMA-targeting CAR-T, JNJ-4528, planned for FDA submission by the end of the year.
In addition to ide-cel and JNJ-4528, BMS is developing its own in-house BCMA-targeting CAR-T for multiple myeloma, orvacabtagene autoleucel.
Photo: CGToolbox, Getty Images

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