EUSA Pharma’s targeted cancer immunotherapy, QARZIBA®▼ (dinutuximab beta) has been approved across Scotland for children with high-risk neuroblastoma.
EUSA welcomed the decision by the Scottish Medicines Consortium (SMC) to accept the use of QARZIBA® to treat children with the disease within NHS Scotland.
This aggressive form of neuroblastoma is rare, but is the most common solid tumour of childhood that originates outside of the brain.
Dinutuximab beta is the first medicine in its class to be accepted for NHS use in Scotland to treat the disease and now offers an option that could help some children keep their cancer at bay for longer.
In a post-hoc analysis, dinutuximab beta has been shown to improve overall survival (OS) outcomes compared to historically-treated patients who did not receive immunotherapy as part of their care.
For some children with high-risk neuroblastoma, who have not received prior immunotherapy and require maintenance therapy, dinutuximab beta is also used to try and keep this aggressive cancer from returning or progressing.
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 the 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 this aggressive cancer (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).
Dinutuximab beta was assessed by the SMC under its ultra-orphan process, reflecting the small numbers of patients that will likely be eligible for treatment each year in Scotland. The guidance from the SMC is that dinutuximab beta be used 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 (high-dose chemotherapy that kills cells in the bone marrow) and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease.
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.
In Scotland, it is estimated that around seven children each year could be eligible for dinutuximab beta. Children with the disease typically undergo many rounds of complex and intensive treatment, usually comprising several cycles of chemotherapy, surgery, stem cell transplant and radiotherapy.
Tony Heddon, Chair of Neuroblastoma UK, said: “Children battling this disease need all the help they can get and that includes access to treatments that may help improve their condition. We welcome this decision and would like to recognise the efforts made on all sides to ensure a successful outcome. The fact that more children will now be able to potentially benefit from this immunotherapy will provide much-needed reassurance for families affected across Scotland.”
Lee Morley, CEO of EUSA Pharma, said: “While today’s approval highlights that progress is being made, much more still needs to be done to help those children and families affected.”