EU/3/18/2094 - orphan designation for treatment of neuroblastoma

Anti-GD2 monoclonal antibody 3F8 humanised

Overview

On 19 November 2018, orphan designation (EU/3/18/2094) was granted by the European Commission to Y-mAbs Therapeutics A/S, Denmark, for anti-GD2 monoclonal antibody 3F8 humanised (also known as naxitamab) for the treatment of neuroblastoma.

What is neuroblastoma?

Neuroblastoma is a cancer of certain 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.

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 was equivalent to a total of around 57,000 people¹, 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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¹Disclaimer: 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 517,400,000 (Eurostat 2018).

 

What treatments are available?

At the time of designation, several methods were authorised in the EU for the treatment of neuroblastoma, including surgery, chemotherapy (medicines to treat cancer) and radiotherapy (treatment with radiation). The medicine Qarziba (dinutuximab beta) was approved for patients with high-risk neuroblastoma (which has a high chance of coming back).

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with neuroblastoma. Preliminary data showed that patients responded in a similar way to this medicine and to existing treatments, but infusions with the medicine could be shorter and less frequent.

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?

The medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure called GD2 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 is expected to make them a target for the body’s immune system (the body’s natural defences), which then kills the cancer cells.

What is the stage of development of this medicine?

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 of the medicine had been granted in the Unites States for neuroblastoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 October 2018 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.

EU/3/18/2094: Public summary of opinion on orphan designation: Anti-GD2 monoclonal antibody 3F8 humanised for the treatment of neuroblastoma

Reference Number: EMA/769941/2018

Key facts

• Active substance:
  Anti-GD2 monoclonal antibody 3F8 humanised
• Intended use:
  Treatment of neuroblastoma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2094
• Date of designation:
  19/11/2018
• Sponsor:

  Y-mAbs Therapeutics A/S; Agern Allé 11; 2970 Hoersholm; Denmark; Telephone: [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.