EU/3/14/1237

Gastro-entero-pancreatic neuroendocrine tumours (GEP-NETs) are tumours that arise from neuroendocrine cells in the gut. These cells release hormones that control various functions of the digestive system. The symptoms of GEP-NETs depend on where the tumour is located within the gut and on whether it produces excess hormones. Often by the time of diagnosis the tumours have spread to other organs such as the liver.

GEP-NETs are debilitating as they often produce excess hormones that may cause severe symptoms. They are life-threatening if they spread to other organs in the body.

At the time of designation, GEP-NETs affected less than 2.8 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 143,000 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 511,100,000 (Eurostat 2014).

At the time of designation, GEP-NETs were diagnosed using various methods. They included histopathology (examining a tissue under the microscope) and biochemical testing (measuring substances produced by neuroendocrine tumours), as well as imaging methods such as magnetic resonance imaging (MRI) and computer tomography (CT) to visualise the location of the tumour. Somatostatin receptor scintigraphy was a commonly used imaging technique, employing a radioactive tracer to obtain an image. At the time of designation, 111In-DTPA-pentetreotide (Octreoscan) was authorised in the EU for use in scintigraphy.

The sponsor has provided sufficient information to show that the medicinal product might be of significant benefit for patients with GEP-NETs because early studies indicate that it may improve the accuracy of detecting those GEP-NETs that have a certain protein known as 'somatostatin receptors' on their surface (somatostatin receptor positive tumours). This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

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 containing a somatostatin analogue, a substance similar to a natural hormone called somatostatin. Most GEP-NETs have high amounts of somatostatin receptors on their surface. The product is expected to attach to these somatostatin receptors and to accumulate in the GEP-NETs cells. These cells are then expected to emit radiation that can be detected by the PET imaging method, thereby allowing the tumour to be diagnosed.

At the time of submission of the application for orphan designation, the evaluation of the effects of the product in experimental models was ongoing.

At the time of submission, no clinical trials with the product in patients with GEP-NETs had been started.
At the time of submission, the product was not authorised anywhere in the EU for diagnosing GEP-NETs or designated as an orphan medicinal product elsewhere for this indication.

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