EU/3/02/085: Orphan designation for the treatment of glioma

Tumours that begin in brain tissue are known as primary brain tumours. Primary brain tumours are classified by the type of tissue from which they originate. The most common brain tumours are gliomas, which begin in the glial (supportive) tissue.

Due to their localisation, gliomas represent a potentially debilitating and life-threatening condition. Patients affected by gliomas can suffer from neurological complications, depending on the site of intra-cerebral development of the tumour.

At the time of designation, gliomas affected approximately 0.85 in 10,000 people in the European Union (EU). This was equivalent to a total of around 32,000 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. At the time of designation, this represented a population of 380,600,000 (Eurostat 2002).

Treatment for gliomas depends on a number of factors and may include surgery, radiotherapy or chemotherapy as well as symptomatic treatments, such as corticosteroids to control the effects of raised intracranial pressure, and anticonvulsants to help control seizures, as required. Methods of treatment of the condition had been authorised at the time of submission of the application for orphan designation. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that carmustine (solution for intratumoural injection) might be of potential significant benefit for the treatment of gliomas, particularly in terms of its new route of administration.

Carmustine is an anti-neoplastic medicinal product. Administration of this product blocks cell multiplication and consequently inhibits tumour growth. The product is administered directly into the brain tumour.

At the time of submission of the application for orphan designation, clinical trials in patients with inoperable recurrent malignant gliomas were ongoing.

Carmustine solution for intratumoural injection had not been marketed anywhere worldwide for gliomas, at the time of submission. Carmustine solution for intratumoural injection has been granted orphan drug designation by the United States Food and Drug Administration for the treatment of intracranial malignancies on 3 July 2000.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 December 2001 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.