The companies resubmitted their application for review to the US Food and Drug Administration (FDA) in July after the agency refused to file it in May, with it requiring more detail in the application’s chemistry, manufacturing and control, or CMC module, in order to complete its review.
But the therapy could be in line for approval by March next year, according to a statement from the partners released this week.
The FDA has given priority review to their resubmitted biologics license application (BLA), they said.
Priority Review
Bluebird said it and BMS requested priority review as part of the July 2020 BLA Submission. On September 22, 2020, the companies announced that the FDA granted that and set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021.
According to the FDA, a priority review designation will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications. Priority review designation also shortens the review time from 12 months to 8 months compared to standard review.
The partners’ investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy is for the treatment of adult patients with multiple myeloma (MM) who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.
Their BLA is built on the results of the Phase II KarMMa study, the results of which were presented at the 2020 American Society of Clinical Oncology meeting.
“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 oncology, bluebird bio.
EU regulatory status
In terms of the regulatory status of the CAR-T therapy in the EU, a spokesperson for BMS reported that the European Medicines Agency (EMA) validated a marketing authorization application (MAA) for ide-cel for myeloma in May 2020 and has begun its centralized review process.
The EMA granted ide-cel accelerated assessment status in March 2020, thus reducing the maximum timeframe for review of the application to 150 days, she said.
Ide-cel was also previously granted PRIority MEdicines (PRIME) designation and validation of its MAA by the EMA for relapsed and refractory multiple myeloma, noted the representative.
“Beyond the US and the EU, we remain committed to making ide-cel available to patients in need across global markets and will provide updates, as appropriate,” she told BioPharma-Reporter.
Retooled deal
BMS picked up the partnership deal with bluebird bio as a result of its acquisition of Celgene at the start of last year.
And, in May this year, the partners announced that they had revised terms of the deal whereby bluebird bio would receive US$200m (€183m) in return for the forfeiture of future ex-US milestone payments and royalties.
Bluebird bio said then that the new arrangements would allow it to ‘refocus’ resources on its internal programs and pipeline.
BMS has now sole responsibility for commercialization and manufacturing of ide-cel ex-US.
Treatment approach
A spokesperson for bluebird bio told us should ide-cel receive approval from the FDA, the partners are committed to making it available as quickly as possible through a network of treatment centers that are appropriately certified and trained to administer CAR-T cell therapies.
"Additionally, the Centers for Medicare and Medicaid Services (CMS) have proposed new policies that would create a new diagnostic related group (DRG) for CAR-T cell therapies in order to promote access and support Medicare reimbursement for these therapies, including ide-cel," she said.
This article was updated on September 25 to include the comments from the bluebird bio spokesperson.
