Overview

On 27 February 2017, orphan designation (EU/3/17/1839) was granted by the European Commission to Y-mAbs Therapeutics A/S, Denmark, for iodine (131I) murine IgG1 monoclonal antibody against CD276 (also known as 131I-mu8H9) for the treatment of 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.

At the time of designation, neuroblastoma affected approximately 0.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 10,000 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 515,700,000 (Eurostat 2017).

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 Unituxin (dinutuximab) was approved for the treatment of children at high risk of the disease returning.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with neuroblastoma because early results in patients with neuroblastoma that has spread or not responded to previous treatments suggest that it can improve survival.This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

The medicine consists of an antibody that has been designed to recognise and attach to a protein called CD276, which is produced in large amounts on the surface of neuroblastoma cells but is not produced in significant amounts by normal tissue. This antibody is linked to radioactive iodine (iodine-131) that produces low-level radiation with a short range.

The medicine is given into the fluid that surrounds the brain and spinal cord, enabling it to reach disease that has spread into the nervous system. When the medicine attaches to cancer cells, radiation from the iodine damages their DNA, resulting in death of the cell. This helps shrink the tumour and control 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, this medicine was not authorised anywhere in the EU for neuroblastoma. Orphan designation of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 January 2017 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.

EU/3/17/1839: Public summary of opinion on orphan designation: Iodine (131I) murine IgG1 monoclonal antibody against CD276 for the treatment of neuroblastoma

AdoptedReference Number: EMA/69663/2017

English (EN) (137.94 KB - PDF)

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Key facts

Active substance
Iodine (131I) murine IgG1 monoclonal antibody against CD276
Intended use
Treatment of neuroblastoma
Orphan designation status
Positive
EU designation number
EU/3/17/1839
Date of designation
Sponsor

Y-mAbs Therapeutics A/S
Agern Allé 11
2970 Hoersholm
Denmark
Telephone: +45 7026 1414
E-mail: info@ymabs.com

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product it 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:

  • 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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