Trade name Kymriah

A first-of-its-kind chimeric antigen receptor T-cell (CAR T) therapy, for the treatment of children and young adults up to age 25 years with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.

An autologous immunocellular therapy consisting of CD3 T cells that have undergone ex-vivid T-cell activation, gene modification, expansion, and formulation infusible cryomedia.

Engineered T-cell therapy that uses a chimeric antigen receptor (CAR) to redirect the cytotoxic machinery of a T cell toward an antigen-expressing tumor cell.

Effective for relapsed/refractory diffuse large B-cell lymphoma, high-grade B-cell lymphoma,and follicular lymphoma-transformed DLBCL.

The transgene expressed via lentiviral vector transduction is a CAR targeted against the CD19 antigen.

Anti-CD19 CAR T cell therapy.

CD19 is a protein is expressed on the surface of B cells from early stages of development and is expressed on most B-cell malignancies.

Has a costimulatory domain of 41BB (CD137).

In a trial of 63 pediatric patients with relapse/refractory ALL-complete remissions occurred in more than 90% of patients.

Within 3 months of treatment 63% of patients achieved complete remission with minimal disease negativity.

Tisagenlecleucel]], in relapsed/refractory diffuse large B cell lymphoma in adults has high rates of durable responses.

Fatal adverse events including infection (4%) and intracerebral hemorrhage (1%), cytokine release syndrome, hypogammaglobulinemia, pyrexia, headache, encephalopathy, hypotension, thrombocytopenia, neutropenia, edema, headache and bleeding can occur.

Tocilizumab (Actemra) can be used to treat CAR T cell–induced severe or life-threatening cytokine release syndrome (CRS), which is a potentially life-threatening side effect of tisagenlecleucel, in patients aged 2 years or older.

Tocilizumab was shown to resolve cytokine release syndrome within 2 weeks after 1-2 doses in 69% of patients.

Due to the cytokine release syndrome risk and risk of neurological events, the approval requires a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use.

CAR-T cell therapies are manufactured for each individual patient, extracting a patient’s T cells to reprogram them outside of the body to recognize and fight specific cells, including cancer cells.

The first-ever gene therapy approved in the United States.

The T cells are genetically modified to include a chimeric antigen receptor that directs the T cells to target and kill leukemia cells with CD19 surface antigen, and are then infused back into the patient.

In a phase 2 clinical trial, the overall remission rate with tisagenlecleucel therapy was 83% in 63 children and young adults with relapsed/refractory B-cell precursor ALL for whom at least two prior lines of therapy had failed.

In phase 2 JULIET trial in refractory and relapsed DLBCL overall response rate was 50%, with 32% and 18% of patients reaching complete response and partial response, respectively.

The efficacy and safety of tisagenlecleucel for the treatment of diffuse large B-cell lymphoma (DLBCL) in the real-world setting are comparable to the efficacy and safety of the pivotal JULIET trial, according to a recent study.

In the JULIET trial, 115 patients received tisagenlecleucel for relapsed or refractory DLBCL.

The best overall response rate was 54%, the complete response rate was 40%,  the median duration of response was not reached after a median follow-up of 24 months, and the 24 month progression free survival was 33%.

In the phase 3 BELINDA trial of  relapsed or refractory aggressive diffuse large B cell  with Tisagenlecleucel compared to salvage chemo immunotherapy followed by high-dose therapy in autologous HSCT: Tisagenlecleucel was not superior to standard therapy

Grade 3 or higher cytokine release syndrome (CRS) and neurotoxicity within the first 8 weeks after infusion was noted in 22.6% and 11.3% of patients, respectively.

In the above study grade 3-4 cytokine release syndrome occurred 23% of patients.

ELIANA trial assessed the efficacy tisagenlecleucel in children and adult patients with relapsed or refactory B-cell ALL- efficacy demonstrated 81% had a complete remission.

Priced at $475,000,

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