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.

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.

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.

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.

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.

  • 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

Key facts

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

Orphelia Pharma S.A.S.
85 Boulevard Saint Michel
75005 Paris
France
Tel: +33(0)1 42 77 08 18
E-mail: contact@orphelia-pharma.eu

Review of designation

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

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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