Dispersion for infusion for the treatment of adults with relapsed and refractory multiple myeloma who have already received at least 3 treatments

About the medicinal product

Carvykti contains the active substance ciltacabtagene autoleucel.

It is used 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).

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, Carvykti has been authorised as an orphan drug. The term “orphan drug” is used to refer to important medicines for rare diseases.

Mode of action

Ciltacabtagene autoleucel, the active substance in Carvykti, is an immunotherapy using genetically modified autologous^[1] T 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

Carvykti is a prescription-only medicine.

It is administered intravenously (into a vein) as an infusion and contains 0.5–1.0 × 10^6 CAR-positive viable T cells per kg body weight, with a maximum dose of 1 × 10^8 CAR-positive viable T cells, and is packaged in a patient-specific infusion bag. The recommended treatment comprises a single infusion of Carvykti, which is administered in a qualified treatment centre under the supervision and guidance of a doctor.

Efficacy

The efficacy of Carvykti was investigated in study MMY2001, in which patients with relapsed and refractory multiple myeloma were treated. The participants had previously received at least 3 lines of treatment, including a proteasome inhibitor, an immunomodulator, and an anti-CD38 antibody.

A total of 97 patients received an infusion of Carvykti with a median dose of 0.71 × 10^6 CAR-positive viable T cells/kg body weight.

The primary endpoint of the study was the objective response rate (ORR^[2]), which was 97.9%, with 80.4% of patients achieving stringent complete remission. In this context, remission means the reduction of the illness or its symptoms. The estimated median^[3] duration of response was 21.8 months and progression-free survival (PFS)^[4] was 22.8 months. The study therefore had a high response rate and a promising duration of response in this patient group.

^[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.}

^[4] _{Progression-free survival (PFS): Period between the start of a treatment or a clinical trial and the onset of disease progression or the death of the patient.}

Precautions, undesirable effects & risks

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

The most frequent undesirable effects were a reduced count of certain groups of white blood cells (neutropenia 89%, lymphopenia 36%, and leukopenia 33%), cytokine release syndrome^[5] (83%), anaemia (deficiency of red blood cells) (60%), and thrombocytopenia (reduced platelet count) (60%).

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

^[5] _{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

At the time of authorisation of Carvykti, there were limited treatment options for patients with relapsed and refractory multiple myeloma who had received at least 3 lines of treatment, including a proteasome inhibitor, an immunomodulator, and an anti-CD38 antibody.

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

The studies performed show a 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 are mitigated by close monitoring and timely intervention. Overall, the positive effects of Carvykti on the health and quality of life of patients outweigh the known risks. Based on these findings, Swissmedic has authorised the medicinal product Carvykti, which contains the active substance ciltacabtagene autoleucel, in Switzerland for the treatment of relapsed and refractory multiple myeloma.

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.}