NICE backs Qarziba for children with high-risk neuroblastoma

High-risk neuroblastoma is an aggressive form of neuroblastoma, the most common solid tumour of childhood that originates outside of the brain.

Qarziba (dinutuximab beta) 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.

The decision by NICE "is a vital step forward in the treatment of young children with this aggressive type of cancer," said Dr Juliet Gray, associate professor in Paediatric Oncology at the Cancer Immunology Centre, University of Southampton.

"By harnessing the body’s own immune system, dinutuximab beta has shown it can target and attack this cancer very effectively in some patients. For some children this could mean extra weeks or months with their families, for others it may even lead to them becoming cancer-free for a long period of time."

Tony Heddon, chair of Neuroblastoma UK, said the recommendation "is a bold and forward-thinking decision from NICE and we applaud them, EUSA Pharma and all those across the community who have worked together to make this medicine available.

"This decision offers the hope that these children with high-risk neuroblastoma, may now have a better future in front of them."

The main aim of current treatment is to extend survival. Until 2009, maintenance therapy with isotretinoin was considered standard care in the NHS for people with high-risk neuroblastoma, but since then, Qarziba has been available through a clinical trial and free supply from the company.

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