EU/3/19/2188 - orphan designation for treatment of neuroblastoma

temozolomide

Overview

On 21 August 2019, orphan designation EU/3/19/2188 was granted by the European Commission to ORPHELIA Pharma S.A.S, France, for temozolomide 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.

What treatments are available?

At the time of designation, several methods were used in the EU for treating neuroblastoma, including surgery, chemotherapy (medicines to treat cancer) and radiotherapy (treatment with radiation). The medicine Qarziba (dinutuximab beta) was authorised 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 because early studies found that the condition improved in patients for whom other treatments did not work or had stopped working. 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?

Temozolomide is a cancer medicine that has been authorised in the EU for several years to treat glioma. It belongs to a group of cancer medicines called alkylating agents. In the body, temozolomide is converted into another substance called methyl diazonium ion, which damages the DNA of cells. This stops tumour cells in the brain from reproducing and slows down the growth of the neuroblastoma.

What is the stage of development of this medicine?

The effects of temozolomide have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with temozolomide in patients with neuroblastoma were ongoing.

At the time of submission, temozolomide was not authorised anywhere in the EU for the treatment of neuroblastoma or designated as an orphan medicinal product elsewhere for this condition. Temozolomide was authorised in the EU for the treatment of glioma under the name Temodal.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 18 July 2019, 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/3/19/2188: Public summary of positive opinion for orphan designation of temozolomide for the treatment of neuroblastoma

Reference Number: EMADOC-628903358-1310

Key facts

• Active substance:
  temozolomide
• Intended use:
  Treatment of neuroblastoma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/19/2188
• Date of designation:
  21/08/2019
• Sponsor:

  Orphelia Pharma S.A.S.; 85 Boulevard Saint Michel; 75005 Paris; France; 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.