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NICE backs EUSA Pharma’s Qarziba for children with high-risk neuroblastoma

The National Institute for Health and Care Excellence (NICE) has recommended the use of the  EUSA Pharma’s targeted cancer immunotherapy, QARZIBA (dinutuximab beta) to treat children with high-risk neuroblastoma within the NHS in England and Wales. 
The company said that the high-risk neuroblastoma is an aggressive form of neuroblastoma – the most common solid tumour of childhood that originates outside of the brain.

Dinutuximab beta is the first targeted cancer immunotherapy approved for use on the NHS  to treat this disease. It has been shown in a post-hoc analysis to improve overall survival (OS) outcomes compared to historically treated patients who did not receive immunotherapy as part of their care.

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

“Today’s 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.”

Dinutuximab beta is a monoclonal antibody (a type of protein) that binds to a specific target which is overexpressed on neuroblastoma cells, called GD2. This induces dual immune mechanisms that then enable the immune system to lead the destruction of neuroblastoma cancer cells. In the key phase III clinical study (APN311-302), a post hoc comparison of dinutuximab beta, used in the maintenance phase of the first-line treatment of high-risk neuroblastoma (n=367) , showed  improved survival outcomes, with a 12% improvement in OS rate at three years versus using no immunotherapy in a historical control group of similar patients (n=450). The dinutuximab beta treated patients had an OS rate of around 65% at 5 years versus 50% compared to the historical control group (p=<0.0001).

Tony Heddon, Chair of Neuroblastoma UK commented: “Ensuring that children and families facing high-risk neuroblastoma have access to the medicines and care they need is absolutely critical. Today’s 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.”

On average, every week, two families in the UK will learn that their child has neuroblastoma, with approximately 100 children diagnosed each year. It is the most frequently-occurring solid tumour in infants under the age of one, accounting for around a fifth (22%) of all cancers diagnosed at this age. Children with high-risk disease to whom this approval applies, account for approximately 40% of all neuroblastoma cases. Children with high-risk neuroblastoma typically undergo many rounds of complex and intensive treatment, usually comprising several cycles of chemotherapy, surgery, stem cell transplant and radiotherapy.

The recommendation from NICE within its Final Appraisal Determination (FAD) is that dinutuximab beta be used as an option for treating high-risk neuroblastoma after at least a partial response from induction chemotherapy, followed by myeloablative therapy and stem cell transplant in people aged 12 months and over, if the person has not had previous anti-GD2 immunotherapy.

Lee Morley, CEO of EUSA Pharma added: “Today’s decision is the result of strong collaboration between NICE, EUSA Pharma and the neuroblastoma community, who have each worked tirelessly to ensure that every eligible child has the option to benefit from this potentially life-changing treatment. 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 today’s 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.”

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