NICE approves neuroblastoma immunotherapy drug for NHS use

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NICE has approved a drug to treat children with high-risk neuroblastoma within the NHS in England and Wales.

EUSA Pharma’s Qarziba (dinutuximab beta) is a monoclonal antibody – a type of protein – that recognises and attaches to a tumour-associated carbohydrate structure, called GD2, which is present in high amounts on the surfaces of neuroblastoma cells.

The latter triggers the body’s immune pathways to attack and destroy cancer cells via the patient’s natural killer immune cells. Dinutuximab beta is the first targeted cancer immunotherapy approved for use on the NHS to treat the disease.

Dr Juliet Gray, associate professor in paediatric oncology at the Cancer Immunology Centre, University of Southampton, said: “Today’s decision by NICE is a vital step forward in the treatment of young children with this aggressive type of cancer.

“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.”

High-risk neuroblastoma is an aggressive form of neuroblastoma – it is the most common childhood solid tumour that originates outside the brain. On average, two families in the UK will learn that their child has neuroblastoma every week, with approximately 100 children diagnosed annually.

Patients typically undergo complex and intensive treatment, usually comprising several cycles of chemotherapy, surgery, stem cell transplant and radiotherapy.

NICE recommends that dinutuximab beta, which is administered intravenously, is used to treat high-risk neuroblastoma after at least a partial response from induction chemotherapy, followed by myeloablative therapy and stem cell transplant in those aged 12 months and over, if the patient has not received previous anti-GD2 immunotherapy.

Lee Morley, CEO of EUSA Pharma said, “Our long-standing commitment has always been to secure access to dinutuximab beta for all eligible children with high-risk neuroblastoma, across the UK and [this] decision is a key part of that journey.

“Beyond England and Wales, we are continuing to work closely with the Scottish and Northern Irish health authorities with the aim of making this medicine available in those countries as quickly as possible.”

Dinutuximab beta received European approval in May 2017, first under the brand names dinutuximab beta Apeiron and Dinutuximab beta EUSA and subsequently under its new name, Qarziba, approved by the European Medicines Agency last November.