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Idecabtagene vicleucel manufacturing and clinical value of out-of-specification products in relapsed and refractory

Surbhi Sidana1, Yi Lin2, Andrew J Cowan3

  • 1Blood and Marrow Transplantation and Cell Therapy Division, Stanford University, Palo Alto, CA.

Blood Advances
|March 27, 2026
PubMed

Insights

Manufacturing of idecabtagene vicleucel (ide-cel) CAR T-cell therapy shows high success rates and improved turnaround times. Out-of-specification product demonstrated comparable safety and efficacy in relapsed/refractory multiple myeloma patients.

Area of Science:

  • Oncology
  • Immunotherapy
  • Cellular Therapy

Background:

  • Idecabtagene vicleucel (ide-cel) is an approved CAR T-cell therapy for relapsed and refractory multiple myeloma (RRMM).
  • Out-of-specification (OOS) ide-cel, not meeting commercial release criteria, can be administered via an expanded access protocol (EAP).
  • Understanding manufacturing reliability and OOS product performance is crucial for patient access and treatment.

Purpose of the Study:

  • To evaluate the manufacturing capability, reliability, and timely delivery of ide-cel in the USA over three years post-approval.
  • To assess the safety and efficacy of OOS ide-cel product in RRMM patients through the BB2121-EAP-001 study.

Main Methods:

  • Analysis of manufacturing data for ide-cel from February 2021 to November 2024, including success rates and turnaround times.
  • Evaluation of safety (primary endpoint) and efficacy (ORR, CRR) of OOS ide-cel in RRMM patients (NCT04771078).
  • Patients included had RRMM and received ide-cel after ≥ 2 prior lines of therapy; OOS product criteria included high CD137 activation or low infused dose.

Main Results:

  • Ide-cel manufacturing success rate improved from 95.3% in 2021 to 96.8% in 2024; turnaround time decreased from 31 to 24 days.
  • In the EAP study, 72.3% of patients experienced cytokine release syndrome (mostly grade 1/2) and 16.0% had neurological toxicities (mostly grade 1/2).
  • Overall response rate (ORR) was 75.4% and complete response rate (CRR) was 35.4% for OOS ide-cel.

Conclusions:

  • Ide-cel manufacturing is reliable with consistently high success rates and improved efficiency.
  • Out-of-specification ide-cel product demonstrates consistent clinical efficacy and a manageable safety profile in RRMM.
  • These findings support the continued use of ide-cel and its OOS product for eligible RRMM patients.

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