Dispersion for infusion for the treatment of adults with relapsed and refractory multiple myeloma who have already received at least 2 treatments

About the medicinal product

Abecma contains the active ingredient idecabtagene vicleucel.

Abecma was first approved on 20 August 2021 for the treatment of advanced multiple myeloma ("bone marrow cancer") in adults whose multiple myeloma (MM) has not responded to 3 previous treatments (refractory), and who have demonstrated disease progression after the last treatment (relapsed).

The indication extension means that Abecma can already be used to treat relapsed and refractory multiple myeloma in adults whose MM has not responded to 2 previous treatments, including an immunomodulator, a proteasome inhibitor, and an anti-CD-38 antibody, and whose disease has progressed during or within 60 days of the last treatment.

MM is a rare form of cancer that accounts for around 1–2% of all cancers. The frequency of new cases of MM increases with age. Two thirds of new sufferers are aged over 65. The disease is characterised by excessive replication of plasma cells, which are a type of white blood cell responsible for producing antibodies in the body's defence system (immune system). In MM, the plasma cells multiply in an uncontrolled way in the bone marrow and occasionally in other organs as well. This prevents the normal formation of blood cells and can destroy, or disrupt the function of, bones and other organs.

Since multiple myeloma is a rare, life-threatening disease, the indication extension for Abecma has been authorised as an orphan drug. The term “orphan drug” is used to refer to important medicines for rare diseases.

Mode of action

The active ingredient in Abecma, idecabtagene vicleucel, is an immunotherapy using genetically modified autologous^[1] T cells (T lymphocytes, belonging to the white blood cells). This therapy uses the patients’ own T cells that have been modified so that they can recognise and fight B cell maturation antigen (BCMA) on the surface of myeloma cells. When the modified T cells are reintroduced into the body, they bind to the cancer cells, activate, and multiply, thereby helping to destroy these cells.

^[1] _{Autologous: Belonging to the same person, i.e. here the patient's own T cells.}

Use

Abecma is a prescription-only medicine.

Abecma is administered intravenously (into a vein) as an infusion, which is supplied in one or more infusion bags, each containing 260 to 500 x 10^6 CAR-positive viable patient-specific T cells. The recommended treatment comprises a single infusion of Abecma, which is administered in a qualified treatment centre under the supervision and guidance of a doctor.

Efficacy

The efficacy of Abecma for the indication extension was investigated in the ongoing study BB2121-MM-003, in which 254 patients with relapsed and refractory multiple myeloma were treated. The participants had previously received at least 2 to 4 lines of treatment, including a proteasome inhibitor, an immunomodulator, and an anti-CD38 antibody. They also had to have been refractory to the last treatment administered. Patients were treated with either Abecma or a standard therapy in the study.

The primary endpoint of the study was progression-free survival (PFS), assessed by an independent evaluation committee. Objective response rate (ORR^[2]) was one of the key secondary endpoints.

Abecma extended median^[3] progression-free survival to 13.3 months, compared to 4.4 months with standard therapy. This result was independent of whether the patients had previously received 2, 3, or 4 lines of treatment. The objective response rate was significantly higher in patients treated with Abecma at 71.3% versus 41.7% for standard therapies.

^[2] _{ORR (objective response rate) is defined as the percentage of patients who respond to the treatment.}

^[3] _{Median: The value that lies exactly in the middle of a distribution of data is called the median or central value. Half of the data values are always less than the median, the other half are always greater.}

Precautions, undesirable effects & risks

Abecma must not be used in those who are hypersensitive to the active substance or any of the excipients.

The most common undesirable effects were neutropenia (reduced count of certain groups of white blood cells, neutrophil granulocytes) (80%), anaemia (deficiency of red blood cells) (64%), thrombocytopenia (reduced platelet count) (55%), infections (44%), hypophosphataemia (low blood phosphate level) (33%), diarrhoea (33%), leukopenia (general reduction in white blood cells) (32%), hypokalaemia (low blood potassium level) (32%), fatigue (30%), nausea (28%), and headaches (23%).

There is also a risk of cytokine release syndrome (CRS)^[4], which occurred in 85% of patients.

Severe neurological side effects such as confusion, headaches, somnolence, and tremor (movement disorder) were also reported.

All precautions, risks, and other possible undesirable effects are listed in the Information for patients (package leaflet) and the Information for healthcare professionals.

^[4] _{Cytokine release syndrome: Cytokine release syndrome is a systemic inflammatory response to the massive secretion of cytokines (proteins), which activate the white blood cells.}

Why the medicinal product has been authorised

The additional study data submitted provide evidence that the benefit-risk assessment of Abecma is favourable for the treatment of adults with recurrent multiple myeloma who have previously received at least 2 lines of treatment, including a proteasome inhibitor, an immunomodulator, and an anti-CD38 antibody, and who were resistant to their previous treatment.

Abecma enables further treatment for this patient group by offering targeted immunotherapy that eliminates malignant cells using genetically modified autologous T cells.

The study performed shows a rapid and significant response rate and continuing effectiveness, with the majority of patients showing no disease progression over longer periods.

While the medicinal product can have potentially severe side effects, these can generally be controlled with close monitoring and timely intervention. Overall, the positive effects of Abecma on the health and quality of life of patients outweigh the known risks.

Based on these findings, Swissmedic has authorised the indication extension for the medicinal product Abecma, containing the active substance idecabtagene vicleucel, in Switzerland.

Further information on the medicinal product

Information for healthcare professionals:

Information for patients (package leaflet):

Healthcare professionals can answer any further questions.

^{The date of revision of this text corresponds to that of the SwissPAR. New information concerning the authorised medicinal product in question will not be incorporated into the Summary report on authorisation.}

^{Swissmedic monitors medicinal products authorised in Switzerland. Swissmedic initiates the necessary action in the event of newly discovered adverse drug reactions or other safety-relevant signals. New findings that could impair the quality, efficacy, or safety of this medicinal product are recorded and published by Swissmedic. If necessary, the medicinal product information is adapted.}