Idecabtagene vicleucel, sold under the brand name Abecma, is a cell-based gene therapy to treat

multiple myeloma 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 antibody, antibodies. Often, no symptoms are noticed initially. As it progresses, bone ...

. The most common side effects include

cytokine release syndrome In immunology, cytokine release syndrome (CRS) is a form of systemic inflammatory response syndrome (SIRS) that can be triggered by a variety of factors such as infections and certain drugs. It refers to cytokine storm syndromes (CSS) and occur ...

(CRS), infections,

fatigue Fatigue is a state of tiredness (which is not sleepiness), exhaustion or loss of energy. It is a signs and symptoms, symptom of any of various diseases; it is not a disease in itself. Fatigue (in the medical sense) is sometimes associated wit ...

, musculoskeletal pain, and a weakened immune system (

hypogammaglobulinemia Hypogammaglobulinemia is an immune system disorder in which not enough gamma globulins are produced in the blood (thus '' hypo-'' + ''gamma'' + '' globulin'' + '' -emia''). This results in a lower antibody count, which impairs the immune system, ...

). Idecabtagene vicleucel is a B-cell maturation antigen (BCMA)-directed genetically modified

autologous Autotransplantation is the transplantation of organs, tissues, or even particular proteins from one part of the body to another in the same person ('' auto-'' meaning "self" in Greek). The autologous tissue (also called autogenous, autogenei ...

chimeric antigen receptor (CAR) T-cell therapy. 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. Idecabtagene vicleucel was approved for medical use in the United States in March 2021. It is the first cell-based gene therapy approved by the US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) for the treatment of multiple myeloma. It was approved for medical use in the European Union in August 2021.

Medical uses

Idecabtagene vicleucel is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

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 Proteasome inhibitors (INN stem ) are drugs that block the action of proteasomes. Proteasomes are large proteins complexes that are used to break down other proteins. These inhibitors are being studied for the treatment of cancer. Drugs such as ...

, and an anti-CD38 monoclonal antibody. 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. This disease keeps the bone marrow from making enough healthy blood cells, which can result in low blood counts. Myeloma can also damage the bones and the kidneys and weaken the immune system. The exact cause of multiple myeloma is unknown. 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.

Adverse effects

The FDA label for idecabtagene vicleucel carries a

boxed warning In the United States, a boxed warning (sometimes "black box warning", colloquially) is a type of warning that appears near the beginning of the package insert for certain prescription drugs, so called because the U.S. Food and Drug Administratio ...

for cytokine release syndrome (CRS), neurologic toxicity,

hemophagocytic lymphohistiocytosis In hematology, hemophagocytic lymphohistiocytosis (HLH), also known as haemophagocytic lymphohistiocytosis ( British spelling), and hemophagocytic or haemophagocytic syndrome, is an uncommon hematologic disorder seen more often in children than ...

macrophage activation syndrome Macrophage activation syndrome is a severe, potentially life-threatening, complication of several chronic rheumatic diseases of childhood. It occurs most commonly with systemic-onset juvenile idiopathic arthritis (SoJIA). In addition, MAS has be ...

(HLH/MAS), and prolonged

cytopenia Cytopenia is a reduction in the number of mature blood cells. It can have many causes, and commonly occurs in people with cancer being treated with radiation therapy or chemotherapy. Types * Anemia – a reduction of the red blood cells in the bo ...

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

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. Efficacy was evaluated in 100 people who received idecabtagene vicleucel in the dose range of 300 to 460 ×10⁶ CAR-positive T cells. Overall, 72% of people partially or completely responded to the treatment. 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. The US

(FDA) granted the application for idecabtagene vicleucel

breakthrough therapy Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "b ...

and

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

designations. The FDA granted approval of Abecma to Celgene Corporation, a

Bristol-Myers Squibb The Bristol-Myers Squibb Company, doing business as Bristol Myers Squibb (BMS), is an American multinational pharmaceutical company. Headquartered in Princeton, New Jersey, BMS is one of the world's largest pharmaceutical companies and consist ...

Company.

Society and culture

Names

Idecabtagene vicleucel is the international nonproprietary name (INN).

References

External links

* {{DEFAULTSORT:Idecabtagene Vicleucel Drugs developed by Bristol Myers Squibb Cancer treatments Drugs that are a gene therapy Approved gene therapies CAR T-cell therapy Orphan drugs