Monthly Archives: April 2015

Anti-O-acetylated-GD2 monoclonal antibody designated as an orphan drug for the treatment of neuroblastoma by EMA

On 2 March 2015 the European Medicines Agency published its public summary of opinion on orphan designation for OGD2 Pharma chimeric monoclonal antibody to O-acetyl-GD2 antigen for the treatment of neuroblastoma granted by the European Commission on 15 January 2015 (EU/3/14/1416).

“The sponsor has provided sufficient information to show that chimeric monoclonal antibody to O-acetyl-GD2 antigen might be of significant benefit for patients with neuroblastoma because it works in a different way to currently authorised treatments by specifically targeting tumour tissue. In addition, early studies in experimental models showed improved anti-tumour activity. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Chimeric monoclonal antibody to O-acetyl-GD2 antigen is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure (an antigen) called O-acetyl-GD2. O-acetyl-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.”

“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 was equivalent to a total of around 56,000 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).

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).”

For more information: European Medicine Agency