SCEMBLIX (asciminib) - Leucémie myéloïde chronique

 Key points

Approval of reimbursement for “the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine kinase inhibitors (see Section 5.1 of the SPC)”.

Note that, in this indication and at the marketing authorisation dosages, patients with a T315I mutation are not included within the scope of the current marketing authorisation.

Therapeutic improvement?

Therapeutic improvement with respect to bosutinib for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine kinase inhibitors.

Role in therapeutic strategy?

The treatment of Ph+ CML in chronic, accelerated or blast phase is essentially based on tyrosine kinase inhibitors (TKIs).

According to international guidelines, the TKIs indicated as a first-line treatment are first- and second-generation treatments (imatinib, dasatinib, bosutinib, and nilotinib), with dasatinib only being reimbursed in France from the second line of treatment. The choice of TKI must be made according to the treatment objectives, age and comorbidities and must account for the safety profile of the medicinal products available, Note that the NCCN guidelines recommend a preference for second-generation TKIs (dasatinib, bosutinib and nilotinib) for patients with an intermediate or high risk score.

TKIs authorised for first-line treatment may be used for second-line treatment, the choice being based on the same criteria as for the first-line approach, as well as on the toxicity of the TKI used as a first-line treatment and the patient’s mutation status. Furthermore, ponatinib (a third-generation TKI) may also be used from the second line of treatment.

As a third and subsequent line of treatment, there are no specific guidelines as to the therapeutic strategy to adopt. In the absence of an alternative, the choice of a TKI must be guided by the BCR-ABL1 mutation sensitivity profile.

According to European guidelines, allogeneic haematopoietic stem cell (HSC) transplantation is a therapeutic option for patients with CML in chronic phase failing to respond (or with a suboptimal response) to two or more TKIs or potentially carrying the T315I mutation. Patients with a high conversion risk must also be envisaged for allogeneic transplantation. Furthermore, allogeneic HSC transplantation may also be a therapeutic option in patients developing an advanced phase during TKI treatment.

Role of the medicinal product

SCEMBLIX (asciminib) is a third-line or subsequent treatment for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase (Ph+ CML-CP), with no T315I mutation and previously treated with two or more tyrosine kinase inhibitors. In the absence of comparative data, it is not possible to determine the role of SCEMBLIX (asciminib) with respect to ponatinib.

Avis économique

Ce produit a fait l'objet d'un avis économique rendu par la Commission d'évaluation économique et de santé publique le 14 février 2023.  L’avis économique porte sur une indication superposable à celle demandée au remboursement, à savoir les patients adultes atteints de LMC-PC Ph+ précédemment traités par au moins deux inhibiteurs de tyrosine kinase. 

La CEESP n’a pas été en mesure de conclure sur l’efficience du produit, en raison d’une réserve majeure portant sur le type d’analyse soumis par l’industriel et invalidant les résultats de l’évaluation économique.   

L’impact budgétaire associé à l’introduction de l’asciminib représente une augmentation des dépenses de l’assurance maladie dans l’indication de 42%, pour une population totale de 9 108 patients cumulés sur 5 ans. Cet impact budgétaire est susceptible d’être sous-estimé en raison de la population cible retenue, inférieure à celle estimée par la Commission de la Transparence.  

> SCEMBLIX - Avis économique (pdf)