Tisagenlecleucel

Kymriah is indicated for the treatment of pediatric and young adult patients (up to age 25) with B-cell acute lymphoblastic leukemia (ALL) that is refractory or has relapsed after at least two prior lines of therapy, as well as adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

Kymriah is an autologous cellular therapy, meaning it uses the patient's own cells. T cells are collected from the patient's blood and genetically engineered in a laboratory to express a chimeric antigen receptor (CAR) that targets the CD19 protein found on the surface of B cells. These modified CAR-T cells are then infused back into the patient, where they can identify and destroy cancer cells expressing CD19.

Kymriah carries a boxed warning for cytokine release syndrome (CRS) and neurological toxicities, both of which can be life-threatening. Other serious risks include prolonged low blood cell counts, serious infections, and tumor lysis syndrome. Due to these risks, Kymriah must be administered only at certified healthcare facilities with staff trained to recognize and manage these complications. Patients should be monitored closely following infusion, typically for several weeks.