2 Information about tisagenlecleucel

2 Information about tisagenlecleucel

Marketing authorisation indication

Tisagenlecleucel (Kymriah, Novartis) is indicated for treating 'paediatric and young adult patients up to 25 years of age with B‑cell acute lymphoblastic leukaemia that is refractory, in relapse post‑transplant or in second or later relapse'.

Tisagenlecleucel is an immunocellular CAR T‑cell therapy. It contains the patient's own T cells (a type of white blood cell) that have been modified genetically in the laboratory so that they make a protein called chimeric antigen receptor (CAR). CAR can attach to another protein on the surface of cancer cells called CD-19. When tisagenlecleucel is given to the patient, the modified T cells attach to and kill cancer cells, thereby helping to clear the cancer from the body.

Dosage in the marketing authorisation

Treatment with tisagenlecleucel comprises a single‑dose intravenous infusion of tisagenlecleucel. It is intended for autologous use only and at the following dosage:

  • For patients ≤50 kg: 0.2 to 5.0×106 CAR‑positive viable T cells per kg body weight.

  • For patients >50 kg: 0.1 to 2.5×108 CAR‑positive viable T cells (non-weight based).

Price

The list price for tisagenlecleucel is £282,000 per infusion (company submission).

The company has a commercial arrangement. This makes tisagenlecleucel available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.

  • National Institute for Health and Care Excellence (NICE)