EU/3/16/1819 - orphan designation for treatment of medulloblastoma

Humanised IgG1 monoclonal antibody against the receptor-binding site of human placental growth factor

Overview

On 12 January 2017, orphan designation (EU/3/16/1819) was granted by the European Commission to Oncurious NV, Belgium, for humanised IgG1 monoclonal antibody against the receptor-binding site of human placental growth factor (also known as TB-403) for the treatment of medulloblastoma.

What is medulloblastoma?

Medulloblastoma is a type of malignant brain tumour that mainly affects children between the ages of three and eight years. The tumour develops in the cerebellum (a region at the bottom of the brain), but may spread to other parts of the brain. The first symptoms of the disease are usually caused by the increased pressure within the skull, and include nausea (feeling sick) and vomiting, headache, ataxia (inability to co-ordinate muscle movements) and irritability.

Medulloblastoma is a debilitating and life-threatening disease because it severely damages the brain and is associated with poor long-term survival.

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

At the time of designation, medulloblastoma 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 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 28), Norway, Iceland and Liechtenstein. This represents a population of 513,700,000 (Eurostat 2016).

What treatments are available?

At the time of designation, the main treatment for medulloblastoma was surgery to remove the tumour. This was usually followed by radiotherapy (treatment with radiation) in children above three years of age, and chemotherapy (medicines to treat cancer), to reduce the risk of the tumour coming back. Two cancer medicines vincristine and carboplatin were specifically authorised for medulloblastoma in the EU.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with medulloblastoma because laboratory studies indicate that it could improve survival in patients whose illness has not responded to existing treatments or has come back after treatment. 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?

In patients with medulloblastoma, the tumour cells produce high levels of a substance called placental growth factor (PlGF), which is normally only present during pregnancy. PlGF is thought to help the cancer cells survive and grow. This medicine contains a monoclonal antibody, a type of protein that has been designed to attach to receptors for PlGF, preventing PlGF from working. In turn this is expected to reduce the growth and spread of the tumour and improve the patient's survival.

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 this medicine in patients with medulloblastoma were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for medulloblastoma 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 8 December 2016 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/3/16/1819: Public summary of opinion on orphan designation: Humanised IgG1 monoclonal antibody against the receptor-binding site of human placental growth factor for the treatment of medulloblastoma

Adopted Reference Number: EMA/4405/2017

Key facts

• Active substance:
  Humanised IgG1 monoclonal antibody against the receptor-binding site of human placental growth factor
• Medicine name:
  -
• Intended use:
  Treatment of medulloblastoma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1819
• Date of designation:
  12/01/2017
• Sponsor:

  Oncurious NV; Gaston Geenslaan 1; B-3001 Leuven; Belgium; Tel. [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.