Venetoclax (3) – Venclyxto®

• Clinical trials
  • The pivotal study M13-982 is a single-arm, uncontrolled Phase II study investigating the efficacy and safety of venetoclax in patients with CLL who had a 17p deletion.

a) Adult patients with CLL who have a 17p deletion or TP53 mutation and who are not suitable for treatment with a B-cell receptor signalling pathway inhibitor or who have experienced treatment failure

• An additional benefit is not proven.
• To demonstrate additional benefit, the pharmaceutical manufacturer has drawn on the results from relevant patient populations of the pivotal study M13-982 and the supportive study M14-032.
• However, on the basis of the available, unadjusted historical control, no conclusions can be drawn regarding the additional benefit for adult patients with CLL who have a 17p-deletion or TP53 mutation and who are unsuitable for treatment with a B-cell receptor signalling pathway inhibitor or have failed such therapy.
• Even taking into account the minor number of patients and, in particular, the magnitude of the treatment effects described in the historical control with the appropriate comparator therapy, it cannot be ruled out with sufficient certainty that potential differences are largely attributable to systematic bias, for example due to relevant differences between the compared populations.
• Overall assessment
  • Due to their minor statistical power, the evidence presented is not suitable for assessing the additional benefit of venetoclax. Overall, the additional benefit for patient population a) is not proven.

b) Adult patients with CLL who do not have a 17p deletion or TP53 mutation and who have experienced treatment failure both with chemoimmunotherapy and with a B-cell receptor signalling pathway inhibitor

• The additional benefit is not proven.
• To demonstrate additional benefit in this patient population b, the pharmaceutical manufacturer has also drawn on the results for relevant patients from the M14-032 study.
• Due to their minor statistical power, the evidence provided is, on the whole, not suitable for assessing the additional benefit of venetoclax.
• Overall, the additional benefit for adult patients with CLL without a 17p deletion or TP53 mutation, who have experienced treatment failure both with chemo-immunotherapy and with an inhibitor of the B-cell receptor signalling pathway, No proof has been provided.
• Overall assessment
  • Overall, the evidence provided is not sufficient to assess the additional benefit of venetoclax due to its minor statistical power. Consequently, an additional benefit for patient group b) is not proven.

Courtesy translation only, please refer to the German original.