Bristol-Myers Squibb and bluebird bio have announced the resubmission of a new biologics license application (BLA) to the US Food and Drug Administration (FDA) for their CAR-T immunotherapy ide-cel.
The immunotherapy, idecabtagene vicleucel (ide-cel/bb2121), is designed to treat adult patients with relapsed and refractory myeloma.
The companies had originally submitted an application to the FDA back in March, although that was met with a refusal to file letter which asked for additional data before the agency processed and reviewed the application.
According to BMS/bluebird, the new submission provides further details on the chemistry, manufacturing and controls (CMC) module of the application to address the FDA’s concerns around the original BLA submission.
The submission is based on results from the phase 2 KarMMa study of ide-cel, which evaluated the drug at three doses in 128 patients who had received on average six previous therapies.
Among this heavily pre-treated group, ide-cel induced an overall response rate of over 73.4% and a complete response rate of 33%. Remissions in the study lasted on average for 10.6 months, and median progression-free survival reached 8.6 months.
BMS/bluebird could face competition from another CAR-T candidate from Johnson and Johnson, which also targets multiple myeloma.
J&J picked up a breakthrough therapy designation (BTD) from the FDA last year for its candidate, JNJ-4528, after the therapy demonstrated impressive results in a phase 1b/2 study in multiple myeloma patients who had received at least three prior lines of therapy.
JNJ-4528 demonstrated early and deep response in phase 1, in patients with a media of five prior multiple myeloma treatment regimens. Impressively, 100% of patients achieved remission at the six-month follow-up.
Overall response rates included 69% of patients achieving a complete response or better, with 86% of patients achieving a very good partial response or better. In addition, 100% of evaluable patients achieved early minimal residual disease-negative status at day 28 post-infusions, which means that there was no detectable cancer cells in their bone marrow.
Across the pond, BMS/bluebird have also been granted an accelerated assessment of ide-cel by the European Medicines Agency (EMA), with the marketing application having been validated in May.
Like J&J’s candidate, ide-cel scored a BTD from the FDA, which will allow for the expedited development and review of the drug now that a comprehensive application has been submitted for review.