EU/3/07/525: Orphan designation for the treatment of neuroblastoma

Neuroblastoma is a life-threatening condition which belongs to a wide group of tumours called neuroendocrine tumours. Neuroblastoma is the most common malignant intra-abdominal tumour in children. Neuroblastoma is often present at birth but is most often diagnosed later when the child begins to show symptoms of the disease. Neuroblastoma manifests as a lump or a mass in the abdomen or around the spinal cord.

At the time of submission of the application for orphan drug designation, removal of the tumour by surgery and radiotherapy was used. Chemotherapy acting both on the tumour and on the metastases was also used. Several products with chemotherapeutic activity were authorised for the condition in some countries in the Community at the time of submission of the application for orphan drug designation.

Iodine (¹³¹I) iobenguane may be of potential significant benefit for the treatment of neuroblastoma in particular with regards to improved efficacy. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

Based on the information provided by the sponsor and previous knowledge of the Committee, neuroblastoma was considered to affect approximately 1.3 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 65,000 persons in total.

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

Iodine (¹³¹I) iobenguane contains a radioactive form of a chemical called Iodine (¹³¹I) and a substance called iobenguane. Iobenguane is similar to the naturally occurring stress hormone noradrenaline and it can specifically be taken up and accumulated by tissues originating in embryonic neural crest cells, such as neuroblastoma tumours. The product is expected to bind to the tumour cells and to kill them with the radiation from the iodine component. The product is a new formulation.

The effects of iodine (¹³¹I) iobenguane were evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with neuroblastoma were initiated.

Iodine (¹³¹I) iobenguane was not authorised anywhere worldwide for the treatment of neuroblastoma, at the time of submission.

Orphan designation of iodine (¹³¹I) iobenguane was granted in the United States for treatment of neuroendocrine tumours.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 5 December 2007 a positive opinion recommending the grant of the above-mentioned designation.

• the seriousness of the condition,
• the existence or not of alternative methods of diagnosis, prevention or treatment and
• either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.