EU regulators have issued a green light for the first immunotherapy for high-risk neuroblastoma, offering thousands of children affected by the rare and devastating form of cancer a new treatment option.
EUSA Pharma’s dinutuximab beta is a monoclonal chimeric antibody developed to target a specific antigen, GD2, on neuroblastoma cells, now indicated to treat the condition in children aged 12 months and above.
According to EU regulators, the drug offers an improvement in the survival of patients when compared with historical controls, while the common side effects were found to be pyrexia, pain and allergic reactions.
Every year in Europe around 1,200 children are diagnosed with neuroblastoma, a cancer arising from neural crest cells, which are involved in the foetal development of the nervous system and other tissues. Almost half of children are diagnosed at an advanced stage of their disease and are considered ‘high-risk’ with a poor prognosis.
According to EUSA, approval of dinutuximab beta brings new hope to these ‘high-risk’ children who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as those with history of relapsed or refractory neuroblastoma, with or without residual disease.
The drug’s approval means that the firm can now make it available for use by hospitals across Europe, improving access for thousands of children and their families to this new treatment, with proven improved survival rates, noted Steve Richards, chief executive of the neuroblastoma charity Solving Kids’ Cancer Europe.
“The next challenge will be for EUSA Pharma to engage with relevant access bodies throughout Europe, including NICE in the UK, to ensure timely review through the new drugs processes and secure access to this medicine for patients. The young innocent victims of this cruel and devastating disease deserve nothing less.”
EUSA, which acquired exclusive global commercialisation rights to the drug from Apeiron Biologics in October last year, said it also expects to file the product for registration in the US and Japan in 2017.