EU/3/16/1794

Gastrointestinal stromal tumours (GISTs) belong to a group of cancers of the stomach and bowel called sarcomas, which are characterised by uncontrolled growth of cells in the supporting tissues of these organs. GISTs are most common in the stomach (60%), followed by the small intestine (30%), and then the colon and rectum (5%). GISTs occur predominantly in middle-aged and older persons, and are considered a life-threatening condition because the tumours could come back and also spread to other organs.

At the time of designation, the number of patients expected to use the medicine for diagnosis of GISTs was approximately 0.15 in 10,000 people in the European Union (EU). This was equivalent to a total of around 8,000 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).

At the time of designation, no medicines were authorised in the EU for the diagnosis of GIST. Non-specific diagnostic methods such as computer tomography (CT), magnetic resonance imaging (MRI) and endoscopy were used.

This medicine is to be used for an imaging method called positron emission tomography (PET). It consists of a radioactive element, gallium (⁶⁸Ga), attached to a substance similar to a 'gastrin releasing peptide'. Most GISTs have high amounts of receptors for gastrin releasing peptide on their surface. By attaching to these receptors, the medicine is expected to build up in the GIST cells and the radioactive substance in the medicine can be detected by PET. This helps to determine the tumours' location and if they have spread to other parts of the body.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with GISTs had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for the diagnosis of GISTs 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 4 November 2016 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.