Idecabtagene vicleucel

Last updated

Idecabtagene vicleucel
Gene therapy
Target gene TNFRSF17
Clinical data
Trade names Abecma
Other namesbb2121, ide-cel
AHFS/Drugs.com Monograph
MedlinePlus a621024
License data
Routes of
administration 		Intravenous
Drug class Antineoplastic
ATC code
Legal status
Legal status
Identifiers
UNII
KEGG

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

Contents

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]

Related Research Articles

<span class="mw-page-title-main">Multiple myeloma</span> Cancer of plasma cells

Multiple myeloma (MM), also known as plasma cell myeloma and simply myeloma, is a cancer of plasma cells, a type of white blood cell that normally produces antibodies. Often, no symptoms are noticed initially. As it progresses, bone pain, anemia, renal insufficiency, and infections may occur. Complications may include hypercalcemia and amyloidosis.

In biology, chimeric antigen receptors (CARs)—also known as chimeric immunoreceptors, chimeric T cell receptors or artificial T cell receptors—are receptor proteins that have been engineered to give T cells the new ability to target a specific antigen. The receptors are chimeric in that they combine both antigen-binding and T cell activating functions into a single receptor.

<span class="mw-page-title-main">B-cell maturation antigen</span> Protein-coding gene in the species Homo sapiens

B-cell maturation antigen, also known as tumor necrosis factor receptor superfamily member 17 (TNFRSF17), is a protein that in humans is encoded by the TNFRSF17 gene.

Elotuzumab, sold under the brand name Empliciti, is a humanized IgG1 monoclonal antibody medication used in combination with lenalidomide and dexamethasone, for adults that have received 1 to 3 prior therapies for the treatment of multiple myeloma. It is also indicated for adult patients in combination with pomalidomide and dexamethasone, who have received 2 prior therapies including lenalidomide and a protease inhibitor. Administration of elotuzumab is done intravenously. Each intravenous injection of elotuzumab should be premedicated with dexamethasone, diphenhydramine, ranitidine and acetaminophen. It is being developed by Bristol Myers Squibb and AbbVie.

<span class="mw-page-title-main">Daratumumab</span> Monoclonal antibody

Daratumumab, sold under the brand name Darzalex among others, is an anti-cancer monoclonal antibody medication. It binds to CD38, which is overexpressed in multiple myeloma cells. Daratumumab was originally developed by Genmab, but it is now being jointly developed by Genmab along with the Johnson & Johnson subsidiary Janssen Biotech, which acquired worldwide commercialization rights to the drug from Genmab.

<span class="mw-page-title-main">Isatuximab</span> Monoclonal antibody

Isatuximab, sold under the brand name Sarclisa, is a monoclonal antibody (mAb) medication for the treatment of multiple myeloma.

<span class="mw-page-title-main">Juno Therapeutics</span> American biopharmaceutical company

Juno Therapeutics Inc was an American biopharmaceutical company founded in 2013 through a collaboration of the Fred Hutchinson Cancer Research Center, Memorial Sloan-Kettering Cancer Center and pediatrics partner Seattle Children's Research Institute. The company was launched with an initial investment of $120 million, with a remit to develop a pipeline of cancer immunotherapy drugs. The company raised $300 million through private funding and a further $265 million through their IPO.

Tisagenlecleucel, sold under the brand name Kymriah, is a CAR T cells medication for the treatment of B-cell acute lymphoblastic leukemia (ALL) which uses the body's own T cells to fight cancer.

Axicabtagene ciloleucel, sold under the brand name Yescarta, is a medication used for the treatment for large B-cell lymphoma that has failed conventional treatment. T cells are removed from a person with lymphoma and genetically engineered to produce a specific T-cell receptor. The resulting chimeric antigen receptor T cells (CAR-Ts) that react to the cancer are then given back to the person to populate the bone marrow. Axicabtagene treatment carries a risk for cytokine release syndrome (CRS) and neurological toxicities.

Lisocabtagene maraleucel, sold under the brand name Breyanzi, is a cell-based gene therapy used to treat B-cell lymphoma and follicular lymphoma.

<span class="mw-page-title-main">Selinexor</span> Anti-cancer drug

Selinexor sold under the brand name Xpovio among others, is a selective inhibitor of nuclear export used as an anti-cancer medication. It works by blocking the action of exportin 1 and thus blocking the transport of several proteins involved in cancer-cell growth from the cell nucleus to the cytoplasm, which ultimately arrests the cell cycle and leads to apoptosis. It is the first drug with this mechanism of action.

bluebird bio, Inc., based in Somerville, Massachusetts, is a biotechnology company that develops gene therapies for severe genetic disorders.

Brexucabtagene autoleucel, sold under the brand name Tecartus, is a cell-based gene therapy medication for the treatment of mantle cell lymphoma (MCL) and acute lymphoblastic leukemia (ALL).

Belantamab mafodotin, sold under the brand name Blenrep, is a monoclonal antibody conjugated with a cytotoxic agent for the treatment of relapsed and refractory multiple myeloma.

Ciltacabtagene autoleucel, sold under the brand name Carvykti, is an anti-cancer medication used to treat multiple myeloma. Ciltacabtagene autoleucel is a BCMA -directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. Each dose is customized using the recipient's own T-cells, which are collected and genetically modified, and infused back into the recipient.

Mosunetuzumab, sold under the brand name Lunsumio, is a monoclonal antibody used for the treatment of follicular lymphoma. It bispecifically binds CD20 and CD3 to engage T-cells. It was developed by Genentech.

Teclistamab, sold under the brand name Tecvayli, is a human bispecific monoclonal antibody used for the treatment of relapsed and refractory multiple myeloma. It is a bispecific antibody that targets the CD3 receptor expressed on the surface of T-cells and B-cell maturation antigen (BCMA), which is expressed on the surface of malignant multiple myeloma B-lineage cells.

Talquetamab, sold under the brand name Talvey, is a humanized monoclonal antibody used for the treatment of multiple myeloma. It is a bispecific GPRC5D-directed CD3 T-cell engager. Talquetamab is a bispecific antibody against two targets: human CD3, a T-cell surface antigen, and human G-protein coupled receptor family C group 5 member D (GPRC5D), a tumor-associated antigen with potential antineoplastic activity. Talquetamab binds both targets, drawing the T cells close to the tumor cells, causing a cytotoxic T-lymphocyte response. It is being developed by Janssen Pharmaceuticals.

Elranatamab, sold under the brand name Elrexfio, is a medication used for the treatment of multiple myeloma. Elranatamab is a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager. Elranatamab is given by subcutaneous injection.

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. 1 2 3 "Abecma- idecabtagene vicleucel suspension". DailyMed. 29 July 2024. Retrieved 20 September 2024.
  4. 1 2 3 4 5 6 7 8 9 10 11 12 "Abecma". U.S. Food and Drug Administration (FDA). 27 March 2021. Archived from the original on 9 December 2023. Retrieved 29 March 2021.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  5. 1 2 "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. 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 "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.PD-icon.svg 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.PD-icon.svg 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

This page is based on this Wikipedia article
Text is available under the CC BY-SA 4.0 license; additional terms may apply.
Images, videos and audio are available under their respective licenses.