EU/3/12/1062: Orphan designation for the treatment of neuroblastoma

Neuroblastoma is a cancer of nerve cells which is usually seen as a lump in the abdomen or around the spine. Symptoms may include weakness, bone pain, loss of appetite and fever.

Neuroblastoma is the most common solid tumour outside the brain in children. In many cases it is present at birth but is diagnosed later when the cancer has spread to other parts of the body and the child begins to show symptoms of the disease.

Neuroblastoma is a long-term debilitating and life-threatening disease that is associated with poor long-term survival.

At the time of designation, neuroblastoma affected approximately 1.1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 51,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000.

This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

At the time of designation, several medicines were authorised in the EU for the treatment of neuroblastoma. Treatments for neuroblastoma included surgery, chemotherapy (medicines to treat cancer) and radiotherapy (treatment with radiation).

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with neuroblastoma because it works in a different way to existing medicines and early studies show that it might improve the outcome of patients with this condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Chimeric monoclonal antibody against GD2 is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure (an antigen) called GD2. GD2 is a substance that is present in high amounts on the surface of neuroblastoma cells, but not normal cells.

When the medicine attaches to the neuroblastoma cells, it marks them out as a target for the body's immune system, which is then expected to attack the cancer cells and thereby reverse or slow down the progression of the disease.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with neuroblastoma were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for neuroblastoma. Orphan designation has been granted in the United States of America for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 October 2012 recommending the granting of this designation.

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

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