EU/3/11/879 - orphan designation for treatment of neuroblastoma

Chimeric monoclonal antibody against GD2 (dinutuximab)

Overview

On 21 June 2011, orphan designation (EU/3/11/879) was granted by the European Commission to United Therapeutics Europe Ltd, United Kingdom, for chimeric monoclonal antibody against GD2 for the treatment of neuroblastoma.

Chimeric monoclonal antibody against GD2 was authorised in the EU as Unituxin on 14 August 2015.

This medicine is now known as dinutuximab.

The marketing authorisation was withdrawn on 20 March 2017.

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products by the European Commission in March 2017 at the time of withdrawal of the marketing authorisation.

What is neuroblastoma?

Neuroblastoma is a cancer of nerve cells which is usually seen as a lump in the abdomen (belly area) 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 life-threatening disease that is associated with poor long-term survival.

What is the estimated number of patients affected by the condition?

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 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).

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*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).

What treatments are available?

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

The sponsor has provided sufficient information to show that chimeric monoclonal antibody against GD2 might be of significant benefit for patients with neuroblastoma because it works in a different way to existing treatments, 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.

How is this medicine expected to work?

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. The way this medicine works is not fully understood, but studies have shown that in the presence of certain cells or proteins of the body's defence system, the neuroblastoma cells to which the medicine is attached die.

What is the stage of development of this medicine?

The effects of chimeric monoclonal antibody against GD2 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 of this medicine had 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 7 April 2011 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria

• 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.
Public

EU/3/11/879: Public summary of opinion on orphan designation: Chimeric monoclonal antibody against GD2 for the treatment of neuroblastoma

AdoptedReference Number: EMA/COMP/300521/2011

Key facts

• Active substance:
  Chimeric monoclonal antibody against GD2 (dinutuximab)
• Medicine name:
  Unituxin
• Intended use:
  Treatment of neuroblastoma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/11/879
• Date of designation:
  21/06/2011
• Sponsor:

  United Therapeutics Europe Ltd; Unither House; Curfew Bell Road; Chertsey; Surrey KT16 9FG; United Kingdom; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

The marketing authorisation was withdrawn on 20 March 2017.

During its meeting of 16 to 18 June 2015, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/11/879 for Unituxin (dinutuximab) 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 maintained¹.

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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 a comparable therapeutic indication cannot be placed on the market.