EU/3/14/1364

On 19 November 2014, orphan designation (EU/3/14/1364) was granted by the European Commission to Noxxon Pharma AG, Germany, for olaptesed pegol for the treatment of glioma.

What is glioma?

Glioma is a type of brain tumour that affects the ‘glial’ cells (the cells that surround and support the nerve cells). Patients with glioma can have severe symptoms, but the types of symptoms experienced depend on where the tumour develops in the brain.

Symptoms can include headaches, nausea (feeling sick), loss of appetite, vomiting, and changes in personality, mood, mental capacity and concentration. About one fifth of patients with glioma have seizures (fits) for months or years before the disease is diagnosed.

Glioma is a long-term debilitating and life-threatening disease because of the severe damage to 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, glioma affected approximately 2.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 112,000 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).

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*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 511,100,000 (Eurostat 2014).

What treatments are available?

At the time of designation, several medicines were authorised for the treatment of glioma in the EU. Treatments included surgery, radiotherapy (treatment with radiation), and chemotherapy (medicines to treat cancer) to improve survival. Patients also received treatments for the symptoms of glioma, including corticosteroids to reduce pressure within the skull and medicines to prevent seizures.

The sponsor has provided sufficient information to show that olaptesed pegol might be of significant benefit for patients with glioma because studies in experimental models showed that the medicine might have improved effects compared with currently available medicines. 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?

Olaptesed pegol works by blocking a protein called stromal cell derived factor-1 (SDF-1), which plays a role in the development of new blood vessels needed by the tumour to grow. By blocking SDF-1, the medicine is expected to prevent the tumour from being well supplied by new blood vessels, eventually killing the tumour cells.

SDF-1 also plays a role in causing the tumour cells to move (metastasise) to other parts of the body. By blocking the action of SDF-1, this medicine is also expected to reduce the spread of the tumour.

What is the stage of development of this medicine?

The effects of olaptesed have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with olaptesed in patients with glioma were planned.

At the time of submission, olaptesed was not authorised anywhere in the EU for glioma. Orphan designation of olaptesed had been granted in the United States for treatment of glioblastoma in conjunction with radiotherapy.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 October 2014 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.

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