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KYMRIAH (tisagenlecleucel), anti-CD19 CAR T

ONCOLOGY - New medicinal product
Opinions on drugs - Posted on Feb 22 2019

Reason for request

Inclusion

High clinical benefit in the third-line or later treatment of refractory or recurrent diffuse large B-cell lymphoma and minor clinical added value in terms of efficacy compared to current management.

 

  •  KYMRIAH has been granted a marketing authorisation for the third-line or later treatment of refractory or recurrent diffuse large B-cell lymphoma (DLBCL).
  • CAR T cells are a gene therapy consisting of autologous T lymphocytes harvested by leukapheresis, then genetically modified ex vivo to express a chimeric receptor antigen targeting protein CD19 found on the B cell line. The re-injected anti-CD19 CAR T cells then multiply and activate in vivo after binding to target cells expressing CD19, thus inducing their apoptosis.

  • Precise quantification of the clinical effect was difficult, failing any comparative studies with usual management, even though direct comparison was possible.

  • In intention-to-treat (ITT) population, the data showed a complete response rate of circa 24.2%, along with an estimated 12-month survival rate of approximately 40%, with a median follow-up period limited to 7 months and adverse events of grade ≥3 in 90% of cases, occasionally requiring hospitalisation in intensive care.

  • Further data are awaited, considering the uncertainties concerning the efficacy, tolerance and complexity of the treatment process.

  • The use of anti-CD19 CAR T is limited to a small number of specifically qualified centres.

 


Actual benefit

Important

-


Improvement in actual benefit

IV (mineur)

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Ce produit a fait l'objet d'un avis d'efficience rendu par la commission évaluation médico-économique et santé publique le 15 janvier 2019.

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