EU/3/14/1416 - orphan designation for treatment of neuroblastoma
Chimeric monoclonal antibody to O-acetyl-GD2 antigen
OrphanHuman
Overview
On 15 January 2015, orphan designation (EU/3/14/1416) was granted by the European Commission to Atlab Pharma SAS, France, for chimeric monoclonal antibody to O-acetyl-GD2 antigen for the treatment of neuroblastoma.
The sponsorship was transferred to OGD2 Pharma, France, in January 2016.
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 isbased 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 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).
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 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.
At the time of submission of the application for orphan designation, the evaluation of the effects of chimeric monoclonal antibody to O-acetyl-GD2 antigen in experimental models was ongoing.
At the time of submission, no clinical trials with the medicine in patients with neuroblastoma had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for neuroblastoma or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 December 2014 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.
Key facts
- Active substance
- Chimeric monoclonal antibody to O-acetyl-GD2 antigen
- Intended use
- Treatment of neuroblastoma
- Orphan designation status
- Positive
- EU designation number
- EU/3/14/1416
- Date of designation
- Sponsor
OGD2 Pharma
3 chemin du Pressoir Chênaie
44100 Nantes
France
Tel. +33 2 28 08 00 31
E-mail: admin@ogd2pharma.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: