22 September 2020 - - The US Food and Drug Administration has accepted for Priority Review US-based biopharmaceutical company Bristol Myers Squibb (NYSE: BMY) and US-based bluebird bio, Inc's (NASDAQ: BLUE) Biologics License Application for idecabtagene vicleucel (ide-cel; bb2121), the companies' investigational B-cell maturation antigen -directed chimeric antigen receptor 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, the companies said.

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021.

The BLA is based on results from the pivotal Phase 2 KarMMa study evaluating the efficacy and safety of ide-cel in 128 adults with heavily pre-treated and highly refractory multiple myeloma exposed to an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.

Results from the study were shared during an oral presentation as part of the American Society of Clinical Oncology 2020 (ASCO20) Virtual Scientific Program.

Ide-cel was granted Breakthrough Therapy Designation by the FDA, and PRIority MEdicines (PRIME) designation and validation of its Marketing Authorization Application by the European Medicines Agency for relapsed and refractory multiple myeloma. Bristol Myers Squibb plans regulatory submissions for ide-cel in additional markets outside the US and EU.

Ide-cel is not approved for any indication in any geography.

US FDA approval of ide-cel by March 31, 2021 is one of the required remaining milestones of the Contingent Value Rights issued upon the close of the Celgene acquisition in the fourth quarter of 2019. The other is US FDA approval of liso-cel by December 31, 2020.

The company is committed to working with FDA to progress both applications and achieve the remaining regulatory milestones required by the CVR.

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with relapsed and refractory multiple myeloma in North America and Europe.

The primary endpoint of the study is overall response rate as assessed by an independent review committee according to the International Myeloma Working Group criteria.

Complete response rate is a key secondary endpoint.

Other secondary endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing assay and safety.

The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells after receiving lymphodepleting chemotherapy.

All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.