ELZONRIS (tagraxofusp)

• Role in the care pathway?

Currently, there is no specific treatment for blastic plasmacytoid dendritic cell neoplasm (BPDCN), and no standardised management. Management is heterogeneous and varies depending on centres and countries.

The treatments used for BPDCN are derived from the chemotherapy protocols used in non-Hodgkin lymphoma (CHOP: cyclophosphamide, doxorubicin, vincristine and prednisone), acute myeloid leukaemia (cytarabine and anthracycline) or acute lymphoblastic leukaemia (Hyper-CVAD: cyclophosphamide, vincristine, doxorubicin, dexamethasone, alternating with methotrexate and cytarabine; or methotrexate and asparaginase).

For eligible patients, and in the event of an identified donor, haematopoietic stem transplant may also be envisaged in the event of a complete response.

Role of the medicinal product in the care pathway

Considering:

• the absence of comparative data enabling assessment of the contribution of ELZONRIS (tagraxofusp) compared to the available alternatives, even though a direct comparative study would have been possible,
• the absence of robust evidence of a benefit on a clinically relevant endpoint,
• the medical need partially met by these alternatives, the use of which is well-established in France,

the Transparency Committee considers that, on the basis of currently available data, ELZONRIS (tagraxofusp) has no role in the care pathway.