A medicine used to treat a rare cancer that predominantly occurs in very young children is to be routinely funded on the NHS in Scotland.

The Scottish Medicines Consortium has endorsed use of EUSA’s Pharma’s Qarziba (dinutuximab beta) to treat children with high-risk neuroblastoma.

Qarziba is a monoclonal chimeric antibody developed to target a specific antigen, GD2, on neuroblastoma cells, approved in Europe last year to treat the condition in children aged 12 months and above.

According to EUSA Pharma, the treatment was shown in a post-hoc analysis to boost overall survival outcomes compared to historically treated patients who did not receive immunotherapy as part of their care.

Qarziba, when used in the maintenance phase of treatment for patients who did not receive prior immunotherapy, is also used to keep condition from returning or progressing in some children with high-risk neuroblastoma.

On the downside, the SMC was unable to accept Roche’s Tecentriq (atezolizumab) for bladder cancer in patients who have already undergone chemotherapy.

Despite the additional flexibility the Patient and Clinician Engagement (PACE) process allows, the Committee was unable to recommend the drug in this setting as there was too much uncertainty in the evidence about both the clinical benefits of the medicine and its cost effectiveness, the cost watchdog noted.

Also not recommended was Biogen’s Fampyra (fampridine) for the improvement of walking in adults with multiple sclerosis (MS), as there was too much uncertainty around its value for money, the SMC said.