EU/3/12/1062 - orphan designation for treatment of neuroblastoma

Chimeric monoclonal antibody against GD2
OrphanHuman

Overview

On 8 November 2012, orphan designation (EU/3/12/1062) was granted by the European Commission to Apeiron Biologics AG, Austria, for chimeric monoclonal antibody against GD2 for the treatment of neuroblastoma.

This medicine is now known as dinutuximab beta.

Chimeric monoclonal antibody against GD2 has been authorised in the EU as Dinutuximab beta EUSA since 8 May 2017.

The sponsorship was transferred to EUSA Pharma (UK) Limited, United Kingdom, in September 2017.

The sponsorship was transferred to EUSA Pharma (Netherlands) B.V, Netherlands, in January 2019.

The sponsor’s address was updated in August 2021.

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.

Key facts

Active substance
Chimeric monoclonal antibody against GD2
Medicine name
Dinutuximab beta Apeiron
Intended use
Treatment of neuroblastoma
Orphan designation status
Positive
EU designation number
EU/3/12/1062
Date of designation
Sponsor

Recordati Netherlands B.V.
Beechavenue 54
Schipol-Rijk
Noord-Holland
1119 PW
Netherlands

E-mail: paulina.skrobuk@eusapharma.com
 

Review of designation

On 18 April 2017, the Committee for Orphan Medicinal Products (COMP) completed its review of the designation EU/3/12/1062 for Dinutuximab beta Apeiron (dinutuximab beta, previously known as chimeric monoclonal antibody against GD2) as an orphan medicinal product for the treatment of neuroblastoma. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with neuroblastoma. The COMP recommended that the orphan designation of the medicine be maintained.1


1The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication cannot be placed on the market.

Update history

DateUpdate
August 2023The sponsor’s name was changed from EUSA Pharma (Netherlands) B.V. to Recordati Netherlands B.V. in August 2023.

Documents related to this orphan designation evaluation

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:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

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:

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