FDA okays first CAR-T cell therapy for a specific myeloma

FDA cleared Bristol Myers Squibb and bluebird bio’s novel CAR-T cell therapy, Abecma (idecabtagene vicleucel; ide-cel). The treatment is the first B-cell maturation antigen (BCMA)-directed CAR-T cell immunotherapy for adults with relapsed or refractory multiple myeloma after 4 or more prior lines of therapy.

Abecma, a personalized immune cell therapy that features a one-time infusion with CAR-positive T cells, carries a wholesale acquisition cost of $419,500. The WAC recognizes “the clinical innovation Abecma represents while maintaining access and affordability,” a spokesperson told FormularyWatch®.

Related: FDA clears BMS CART-T cell therapy, Merck MET inhibitor

A network has been created to support “rapid and dependable manufacturing of Abecma and ensure capacity to accommodate patient demand,” the pharma makers said in a press release. Abecma will be manufactured for each individual patient using their own T cells at BMS’s immunotherapy manufacturing facility in Summit, NJ.

BMS is working to onboard a network of certified treatment centers to begin treating patients with the CAR-T therapy. Treatment centers will be Risk Evaluation and Mitigation Strategy (REMS) certified to support appropriate use of Abecma, which is available only through the Abecma REMS program, the spokesperson said.

As an anti-BCMA CAR T cell therapy, Abecma recognizes and binds to BCMA, a protein that is nearly universally expressed on cancer cells in multiple myeloma, leading to the death of BCMA-expressing cells, BMS and bluebird bio said.

Related: FDA clears first cell-based gene therapy for lymphoma

“CAR T cell therapies have shown transformational potential for the treatment of hematologic malignancies, and we, with our partners at bluebird bio, are proud to bring the first CAR T cell therapy to appropriate triple-class exposed patients with relapsed or refractory multiple myeloma, offering the chance for durable response,” said Samit Hirawat, MD, chief medical officer, Bristol Myers Squibb.

FDA’s approval is based on data from the pivotal Phase 2 KarMMa trial of 127 patients with relapsed or refractory multiple myeloma who had received at least 3 prior lines of therapy including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.

The overall response rate (ORR) for the efficacy evaluable population was 72% and 28% of patients achieved a stringent complete response (sCR). Responses were rapid and durable, with a median time to response of 30 days and median duration of response of 11 months for all responders and 19 months who achieved sCR.

Of the 28 patients who achieved sCR, an estimated 65% had remission lasting at least 12 months.

Read more: A conversation about real-world data with COTA’s Viraj Narayanan