Idecabtagene vicleucel

Idecabtagene vicleucel, sold under the brand name Abecma, is a cell-based gene therapy to treat multiple myeloma.^[3][7][8]

Idecabtagene vicleucel

Gene therapy
Target gene	TNFRSF17
Clinical data
Trade names	Abecma
Other names	bb2121, ide-cel
AHFS/Drugs.com	Monograph
MedlinePlus	a621024
License data	US DailyMed: Idecabtagene vicleucel
Routes of administration	Intravenous
Drug class	Antineoplastic
ATC code	L01XL07 (WHO)
Legal status
Legal status	CA: ℞-only / Schedule D[1] US: WARNING[2]Rx-only[3][4]< EU: Rx-only[5][6]
Identifiers
UNII	8PX1X7UG4D
KEGG	D11556

The most common side effects include cytokine release syndrome (CRS), infections, fatigue, musculoskeletal pain, and a weakened immune system (hypogammaglobulinemia).^[4][7]

Idecabtagene vicleucel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy.^[4][7] Each dose is customized using a patient's own T-cells, which are a type of white blood cell, that are collected and genetically modified to include a new gene that facilitates targeting and killing myeloma cells, and infused back into the patient.^[4][7]

Idecabtagene vicleucel was approved for medical use in the United States in March 2021.^[4][7] It is the first cell-based gene therapy approved by the US Food and Drug Administration (FDA) for the treatment of multiple myeloma.^[4][7] It was approved for medical use in the European Union in August 2021.^[5][9]

Medical uses

Idecabtagene vicleucel is indicated for the treatment of adults with relapsed or refractory multiple myeloma after two or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.^[3][4]

Multiple myeloma is an uncommon type of blood cancer in which abnormal plasma cells build up in the bone marrow and form tumors in many bones of the body.^[7] This disease keeps the bone marrow from making enough healthy blood cells, which can result in low blood counts.^[7] Myeloma can also damage the bones and the kidneys and weaken the immune system.^[7] The exact cause of multiple myeloma is unknown.^[7] According to the National Cancer Institute, myeloma accounted for approximately 1.8% (32,000) of all new cancer cases in the United States in 2020.^[7]

Adverse effects

The FDA label for idecabtagene vicleucel carries a boxed warning for cytokine release syndrome (CRS), neurologic toxicity, hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), and prolonged cytopenia.^[4][7] CRS and HLH/MAS are systemic responses to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and prolonged cytopenia is a drop in the number of a certain blood cell type for an extended period of time.^[7]

In April 2024, the FDA label boxed warning was expanded to include T cell malignancies.^[10]

History

The safety and efficacy of idecabtagene vicleucel were evaluated in a multicenter study of 127 people with relapsed (myeloma that returns after completion of treatment) and refractory (myeloma that does not respond to treatment) multiple myeloma who received at least three prior lines of antimyeloma therapies; 88% had received four or more prior lines of therapies.^[4] Efficacy was evaluated in 100 people who received idecabtagene vicleucel in the dose range of 300 to 460 ×10⁶ CAR-positive T cells.^[4] Overall, 72% of people partially or completely responded to the treatment.^[7] Of those studied, 28% of people showed complete response—or disappearance of all signs of multiple myeloma—to idecabtagene vicleucel, and 65% of this group remained in complete response to the treatment for at least twelve months.^[7]

The US Food and Drug Administration (FDA) granted the application for idecabtagene vicleucel breakthrough therapy and orphan drug designations.^[4][7] The FDA granted approval of Abecma to Celgene Corporation, a Bristol-Myers Squibb Company.^[4][7]

Society and culture

Names

Idecabtagene vicleucel is the international nonproprietary name (INN).^[11]

References

1. "Summary Basis of Decision (SBD) for Abecma". Health Canada. 23 October 2014. Archived from the original on 29 May 2022. Retrieved 29 May 2022.
2. "FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 October 2023.
3. "Abecma- idecabtagene vicleucel suspension". DailyMed. 29 July 2024. Retrieved 20 September 2024.
4. "Abecma". U.S. Food and Drug Administration (FDA). 27 March 2021. Archived from the original on 9 December 2023. Retrieved 29 March 2021. This article incorporates text from this source, which is in the public domain.
5. "Abecma EPAR". European Medicines Agency (EMA). 23 June 2021. Archived from the original on 26 August 2021. Retrieved 26 August 2021.
6. "Abecma Product information". Union Register of medicinal products. Archived from the original on 5 March 2023. Retrieved 3 March 2023.
7. "FDA Approves First Cell-Based Gene Therapy for Adult Patients with Multiple Myeloma". U.S. Food and Drug Administration (FDA) (Press release). 27 March 2021. Archived from the original on 28 March 2021. Retrieved 29 March 2021. This article incorporates text from this source, which is in the public domain.
8. "U.S. Food and Drug Administration Approves Bristol Myers Squibb's and bluebird bio's Abecma (idecabtagene vicleucel), the First Anti-BCMA CAR T Cell Therapy for Relapsed or Refractory Multiple Myeloma" (Press release). Bristol Myers Squibb. 26 March 2021. Archived from the original on 29 March 2021. Retrieved 29 March 2021 – via Business Wire.
9. "Abecma". Union Register of medicinal products. 19 August 2021. Archived from the original on 1 October 2023. Retrieved 1 October 2023.
10. "FDA Requires Boxed Warning for T cell Malignancies Following Treatment with BCMA-Directed or CD19-Directed Autologous Chimeric Antigen Receptor (CAR) T cell Immunotherapies". U.S. Food and Drug Administration (FDA). 18 April 2024. Archived from the original on 19 April 2024. Retrieved 19 April 2024. This article incorporates text from this source, which is in the public domain.
11. World Health Organization (2019). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81". WHO Drug Information. 33 (1): 78-9. hdl:10665/330896.

Further reading

• Munshi NC, Anderson LD, Shah N, Madduri D, Berdeja J, Lonial S, et al. (February 2021). "Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma". N Engl J Med. 384 (8): 705–16. doi:10.1056/NEJMoa2024850. hdl:11585/865225. PMID 33626253. S2CID 232048693.{{cite journal}}: CS1 maint: overridden setting (link)
• Raje N, Berdeja J, Lin Y, Siegel D, Jagannath S, Madduri D, et al. (May 2019). "Anti-BCMA CAR T-Cell Therapy bb2121 in Relapsed or Refractory Multiple Myeloma". N Engl J Med. 380 (18): 1726–37. doi:10.1056/NEJMoa1817226. PMC 8202968. PMID 31042825.{{cite journal}}: CS1 maint: overridden setting (link)

External links

• Clinical trial number NCT03361748 for "Efficacy and Safety Study of bb2121 in Subjects With Relapsed and Refractory Multiple Myeloma (KarMMa)" at ClinicalTrials.gov