EU/03/14/1269

On 4 June 2014, orphan designation (EU/03/14/1269) was granted by the European Commission to ITG Isotope Technologies Garching GmbH, Germany, for lutetium (¹⁷⁷Lu) edotreotide for the treatment of gastro-entero-pancreatic neuroendocrine tumours.

What are gastro-entero-pancreatic neuroendocrine tumours?

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.

What is the estimated number of patients affected by the condition?

At the time of designation, GEP-NETs affected approximately 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 179,000 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).

---

*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 the application for orphan designation, several products were authorised in the EU for treating GEP-NETs, including octreotide, everolimus, and sunitinib.

The sponsor has provided sufficient information to show that lutetium (¹⁷⁷Lu) edotreotide might be of significant benefit for patients with GEP-NETs because data showed that patients treated with the medicine lived for longer without their disease getting worse. 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?

Most tumour cells of patients with GEP-NETs have high amounts of receptors for a hormone called somatostatin. Lutetium (¹⁷⁷Lu) edotreotide has two main components, one of which, edotreotide, has a similar structure to somatostatin and can attach to the same receptors on the tumour cells, while the other is a radioactive form of the substance lutetium (¹⁷⁷Lu). By attaching to the tumour cells and then by delivering a small amount of short-range radiation from the lutetium, the medicine is expected to be able to kill the tumour cells, and thereby stop or slow down the progression of the disease.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of lutetium (¹⁷⁷Lu) edotreotidein experimental models was ongoing.

At the time of submission, no clinical trials with the medicine in patients with GEP-NETs had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for GEP-NETs 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 9 April 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.