After receiving a discouraging refuse-to-file letter from the US Food and Drug Administration (FDA) in May, Bristol Myer Squibb and bluebird bio’s submission for their CAR-T immunotherapy ide-cel has finally been accepted by the agency.
BMS and bluebird bio initially submitted an application with the FDA in March, but that was met with a refuse-to-file letter in May which asked the companies for additional data before the agency processed and reviewed the submission.
The companies then announced that they had re-filed the biologics license application (BLA) in July, saying that the new application provided further details on the chemistry, manufacturing and control (CMC) module of the submission to address the FDA’s concerns around the original BLA.
The immunotherapy, idecabtagene vicleucel (ide-cel/bb2121), is designed to treat adult patients with relapsed and refractory myeloma. The FDA submission is based on results from the phase 2 KarMMa study of ide-cel, which evaluated the therapy of three doses in 128 patients who had received on average six previous therapies.
In this heavily pre-treated group, ide-cel induced an overall response rate of 81.5% and a complete response rate of 35.2%. Remissions in the study lasted an average of 10.6 months, and median progression-free survival reached 8.6 months.
BMS and bluebird bio are aiming for approval of ide-cel in adult patients with multiple myeloma who have received at least three prior therapies, including an immunomuodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, such as Janssen’s Darzalex (daratumumab).
“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,” said Joanne Smith-Farrell, chief operating officer of oncology at bluebird bio
“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,” she added.
Following the acceptance of the application and priority review designation, the FDA has set a Prescription Drug User Fee Act (PDUFA) date of 27 March 2021, setting up a potential launch of ide-cel in the second half of next year.
Ide-cel has been awarded a Priority Medicines (PRIME) designation by the European Medicines Agency (EMA), and BMS and bluebird bio also plan to submit the CAR-T therapy for approval in additional markets outside the US and EU soon.