Dataset Information

FDA Approval Summary: Idecabtagene Vicleucel for Relapsed or Refractory Multiple Myeloma.

ABSTRACT: In March 2021, the FDA approved idecabtagene vicleucel, a chimeric antigen receptor T-cell therapy targeting the B-cell maturation antigen (BCMA), for adult patients with relapsed/refractory multiple myeloma (RRMM) after ≥4 lines of therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 mAb. Approval was based on overall response rate (ORR), complete response (CR) rate, and duration of response (DOR) in 100 adult patients with RRMM treated with idecabtagene vicleucel in a single-arm trial. Patients received a single infusion of idecabtagene vicleucel, preceded by lymphodepleting chemotherapy with cyclophosphamide and fludarabine. Of the 100 patients in the efficacy evaluable population, ORR was 72% [95% confidence interval (CI), 62-81] with stringent CR rate of 28% (95% CI, 19-38). After median follow-up of 10.7 months, median DOR was 11 months (95% CI, 10.3-11.4) in responders (partial response or better) and 19 months [95% CI, 11.4 months, not estimable (NE)] in patients who achieved stringent CR. Serious adverse reactions occurred in 67% of 127 patients evaluated for safety. Grade 3 or higher cytokine release syndrome and neurologic toxicities occurred in 9% and 4%, respectively, leading to a Risk Evaluation and Mitigation Strategy. Hemophagocytic lymphohistiocytosis/macrophage activation syndrome occurred in 4%, with two fatalities. Prolonged cytopenia requiring hematopoietic rescue occurred in 2% (3/127), with two fatalities.

SUBMITTER: Sharma P

PROVIDER: S-EPMC9064878 | biostudies-literature | 2022 May

REPOSITORIES: biostudies-literature

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FDA Approval Summary: Idecabtagene Vicleucel for Relapsed or Refractory Multiple Myeloma.

Sharma Poornima P Kanapuru Bindu B George Bindu B Lin Xue X Xu Zhenzhen Z Bryan Wilson W WW Pazdur Richard R Theoret Marc R MR

Clinical cancer research : an official journal of the American Association for Cancer Research 20220501 9

In March 2021, the FDA approved idecabtagene vicleucel, a chimeric antigen receptor T-cell therapy targeting the B-cell maturation antigen (BCMA), for adult patients with relapsed/refractory multiple myeloma (RRMM) after ≥4 lines of therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 mAb. Approval was based on overall response rate (ORR), complete response (CR) rate, and duration of response (DOR) in 100 adult patients with RRMM treated with idecabtagene vicleuc ...[more]

PMID: 35046063

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Project description:Idecabtagene vicleucel (ide-cel), a B-cell maturation antigen-directed chimeric antigen receptor T cell therapy, showed deep, durable responses in patients with triple-class exposed, relapsed and refractory multiple myeloma (RRMM) in the phase 2 KarMMa (Efficacy and Safety Study of bb2121 in Subjects With Relapsed and Refractory Multiple Myeloma) trial. We assessed health-related quality of life (HRQoL) among KarMMa patients. The European Organization for Research and Treatment of Cancer Quality of Life C30 Questionnaire and its supplementary 20-item multiple myeloma module, as well as the EuroQol 5-dimension 5-level instrument, were administered at screening, baseline (≤72 hours before or same day as lymphodepletion), day of ide-cel treatment, and after ide-cel treatment. Mean changes from baseline that exceeded the predetermined threshold of minimally important difference were deemed clinically meaningful. The proportions of patients experiencing clinically meaningful changes in HRQoL were assessed using within-patient change thresholds. Time to stable improvement (≥2 consecutive visits with clinically meaningful HRQoL improvements) was analyzed by using the Kaplan-Meier method. A total of 126 (98%) of 128 patients treated with ide-cel were included in the HRQoL analysis. Pretreatment baseline RRMM burden was high and meaningfully worse than that in the age- and sex-weighted general population. Statistically significant and clinically meaningful improvements from baseline were observed by month 1 for pain (-8.9) and disease symptoms (-10.2), and by month 2 for fatigue (-7.2), physical functioning (6.1), cognitive functioning (6.7), and global health status/QoL (8.0). Clinically meaningful improvements in fatigue, pain, and physical functioning were most prominent at months 9, 12, and 18, respectively, and were sustained through 15 to 18 months after ide-cel treatment. For triple-class exposed patients with RRMM with a poor prognosis and few treatment options, a single ide-cel infusion provides early, sustained, statistically significant, and clinically meaningful improvements in HRQoL. This study was registered at Clinicaltrials.gov as #NCT03361748.

Project description:IntroductionPatients with triple-class exposed relapsed/refractory multiple myeloma (RRMM) have poor outcomes with substantial healthcare costs. Idecabtagene vicleucel (ide-cel), a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell therapy, showed deep, durable responses in patients with RRMM in the pivotal phase 2 KarMMa trial (NCT03361748). Healthcare resource utilization (HCRU) and costs were estimated for ide-cel-treated patients in the KarMMa trial.MethodsPost-infusion costs were estimated based on HCRU data, including facility care, diagnostics, medications, and procedures. Length of stay (LOS) was extracted for inpatient and intensive care unit (ICU) care. All patients had a 14-day post-infusion inpatient stay per trial protocol. Analyses were conducted for patients treated in the United States (US), who received the ide-cel target dose of 450 × 106 CAR + T cells and assuming a 7-day inpatient stay.ResultsOverall, 128 patients received ide-cel and were included in this analysis. Mean age was 60 years, 59% were men, and 81% were white. Mean total LOS was 23.9 days. Total estimated costs over 24 months post-infusion were US$115,614 per patient, driven by facility costs (75%; $86,385). Most costs were incurred in the first month (58%; $67,259). The scenario analysis assuming a 7-day inpatient stay showed estimated 24-month costs of $92,294. For the 54 (42%) patients who received ide-cel high dose, total costs over 24 months were $113,298 per patient.ConclusionsExtrapolation of costs based on HCRU data from patients receiving single-infusion of ide-cel in the KarMMa trial showed substantially reduced HCRU and costs over 2 years after initial treatment. Most costs were incurred during the first month after ide-cel infusion, likely attributable to the 14-day inpatient stay required by the trial protocol. These findings suggest a nominal, incremental monthly cost of care immediately after treatment, which may be lower in routine clinical practice.

Dataset Information

FDA Approval Summary: Idecabtagene Vicleucel for Relapsed or Refractory Multiple Myeloma.

Publications

FDA Approval Summary: Idecabtagene Vicleucel for Relapsed or Refractory Multiple Myeloma.

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