dinutuximab beta (Qarziba)

following a full submission: assessed under the ultra-orphan process

dinutuximab beta (Qarziba®) is accepted for use within NHSScotland.

Indication under review: for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilised by other suitable measures.

In patients with a history of relapsed/refractory disease and in patients who have not achieved a complete response after first line therapy, dinutuximab beta should be combined with interleukin-2.

Comparisons with historical controls indicate that dinutuximab beta plus isotretinoin with and without aldesleukin (interleukin-2) improved event-free survival and overall survival in patients undergoing first-line treatment for high-risk neuroblastoma and improved overall survival in patients with relapsed neuroblastoma. In patients with relapsed or refractory neuroblastoma dinutuximab beta in combination with isotretinoin and aldesleukin was associated with tumour responses.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of dinutuximab beta. This advice is contingent upon the continuing availability of the PAS in NHSScotland or a list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.