The European Commission granted a marketing authorisation valid throughout the European Union for Dinutuximab beta Apeiron on 8 May 2017. It is a orphan medicine and it has been authorised under exceptional circumstances.
This is because it has not been possible to obtain complete information about Dinutuximab beta Apeiron for ethical reasons. As dinutuximab is a recommended treatment for high-risk neuroblastoma, it would not be ethical to carry out a trial in which some patients were given placebo. Dinutuximab beta Apeiron is a cancer medicine used to treat neuroblastoma, a cancer of nerve cells, in patients over 1 year of age. Dinutuximab beta Apeiron is used together with another medicine called interleukin-2 (aldesleukin) in some cases where previous treatments have not worked well enough. The medicine contains the active substance dinutuximab beta.
It is a monoclonal antibody that has been designed to recognise and attach to a structure called GD2 that is present in high amounts on the surface of neuroblastoma cells, but not normal cells. When Dinutuximab beta Apeiron attaches to the neuroblastoma cells, it makes them a target for the body’s immune system, which then kills the cancer cells. Studies have shown that Dinutuximab beta Apeiron is effective at increasing survival in patients with neuroblastoma. Two studies analysed data from 88 children and adults with neuroblastoma that had not improved with other cancer treatments or had come back. Patients were treated with Dinutuximab beta Apeiron plus interleukin-2 and another medicine called isotretinoin. In these studies, 70% and 78% of the patients whose neuroblastoma had not improved with other treatments were still alive 2 years after treatment. Of the patients with neuroblastoma that had come back, 42% and 69% were still alive 2 years after treatment. In a third study, 370 children with high-risk neuroblastoma that had improved after other treatments were given Dinutuximab beta Apeiron and isotretinoin with or without interleukin-2. At the start of treatment some of these patients had no sign of neuroblastoma and some still had some sign of the disease.
Of the patients who had no sign of neuroblastoma, 71% were still alive 3 years after treatment and the results were similar whether the treatment included interleukin-2 or not. Of the patients who had some sign of neuroblastoma, 63% of those given interleukin-2 were still alive 3 years after treatment compared with 54% of patients who did not receive interleukin-2. In these studies, outcomes with Dinutuximab beta Apeiron compared favourably with those previously seen in patients treated for neuroblastoma without Dinutuximab beta Apeiron. The most common side effects with Dinutuximab beta Apeiron are pyrexia and pain, hypersensitivity (allergy), vomiting, diarrhoea, capillary leak syndrome and hypotension.