Regulatory Decision Summary for Abecma
Review decision
The Regulatory Decision Summary explains Health Canada’s decision for the product seeking market authorization. The Regulatory Decision Summary includes the purpose of the submission and the reason for the decision.
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What was the purpose of this submission?
The purpose of this submission was to seek market authorization for Abecma (idecabtagene vicleucel), a B-cell maturation antigen (BCMA)-directed genetically modified autologous 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.
After evaluation of the submitted data package, Health Canada authorized Abecma, with conditions, for the following indication:
Abecma (idecabtagene vicleucel), a B-cell maturation antigen (BCMA)-directed genetically modified autologous T-cell immunotherapy, is indicated 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 and who are refractory to their last treatment.
Why was the decision issued?
The authorization of Abecma (idecabtagene vicleucel) was based on the results of an open-label single-arm study designed to evaluate the efficacy and safety of Abecma in patients with relapsed and refractory multiple myeloma (RRMM).
A total of 140 patients were enrolled on study and 128 patients received treatment with Abecma. The majority of patients received bridging therapy before administration of Abecma and six patients had a complete response (CR) following bridging therapy. The efficacy analyses were based on the 122 patients who had measurable disease at the time of Abecma infusion.
The primary efficacy endpoint on study was the Overall Response Rate (ORR). Important secondary endpoints were combined stringent complete response (sCR) and CR (sCR + CR), very good partial response and duration of response (DOR). Seventy-four percent of patients had an ORR and thirty-two percent of patients had a sCR or CR. The median DOR for ORR was 11 months and the median DOR for combined sCR + CR was 19 months. For patients who had a response to therapy, the median time to response was 1 month from the Abecma infusion.
The safety profile of Abecma is similar to other authorized CAR T-cell therapies and includes serious adverse reactions, most notably cytokine release syndrome (CRS) and neurologic toxicities. Another serious event on study was hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS). Clinical features of HLH/MAS include persistent high-grade fevers, hepatosplenomegaly, lymphadenopathy, hemorrhagic manifestations, and sepsis-like conditions. Other important toxicities related to Abecma were hypogammaglobulinemia (increased risk of infection), hypersensitivity reactions, serious infections (including fatalities), viral reactivations, and prolonged cytopenias.
The recommended dose is a single infusion of Abecma between 275 to 520 x 106 CAR+ T-cells with a target dose of 450 x 106 CAR+ T-cells. Refer to the Product Monograph for details.
Overall, the benefit-risk profile is considered positive when Abecma is administered at qualified treatment centres where the risks of this therapy can be properly monitored and managed. Abecma is considered a promising treatment option for multiple myeloma patients who have received multiple prior therapies. Health Canada has requested that the benefit of Abecma in patients with relapsed and refractory multiple myeloma be confirmed in the ongoing Phase III KarMMa3 study.
A Notice of Compliance with Conditions (NOC/c) was recommended.
Decision issued
Approved; issued a Notice of Compliance in accordance with the Food and Drug Regulations