EU/3/08/589 - orphan designation for treatment of gastro-entero-pancreatic neuroendocrine tumours

yttrium (90Y) edotreotide

Overview

On 4 December 2008, orphan designation (EU/3/08/589) was granted by the European Commission to Molecular Insight Pharmaceuticals GmbH, Germany, for yttrium (90Y) edotreotide for the treatment of gastro-entero-pancreatic neuroendocrine tumours.

The sponsorship was transferred to Molecular Insight Limited, United Kingdom, in June 2010 then to ITG Isotope Technologies Garching GmbH, Germany, in July 2013 and back to Molecular Insight Limited, United Kingdom, in January 2016.

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in April 2018 on request of the Sponsor.

What are gastro-entero-pancreatic neuroendocrine tumours?

Gastro-entero-pancreatic neuroendocrine tumours (GEP-NETs) are tumours of the 'neuroendocrine system' in the gut. This is the system where the nervous system and hormonal system interact to control the digestive organs. These tumours share a number of common characteristics, for example content of specific chemical substances called 'neuroendocrine markers'. There are two main types of GEP-NETs: carcinoid tumours, and 'pancreatic-type' endocrine tumours (these often occur in the pancreas, but also in other sites). GEP-NETs are debilitating as they often secrete 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, gastro-entero-pancreatic neuroendocrine tumours affected approximately 1.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 80,000 people*, and is below the threshold 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).

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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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 502,800,000 (Eurostat 2008).

What treatments are available?

At the time of submission of the application for orphan designation, several medicines were authorised in the EU for the treatment of one or more types of GEP-NET. In most cases, these medicines act on the symptoms caused by the secretion of hormones, but are not active against the growth of the tumours. Commonly, surgery to remove the tumours and treatment with 'somatostatin analogues' were used. Somatostatin analogues are similar to a naturally occurring hormone somatostatin that prevents the release of many hormones.

The sponsor has provided sufficient information to show that yttrium (⁹⁰Y) edotreotide might be of significant benefit for patients because it is expected to kill tumour cells, reduce tumour size and eventually improve the long term outcome of the patients. This assumption will have to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Yttrium (⁹⁰Y) edotreotide contains a somatostatin analogue called edotreotide that has been tagged with a radioactive form of the chemical element yttrium called yttrium-90 (⁹⁰Y). The somatostatin analogue is expected to attach to the tumour cells, where the radiation from the ⁹⁰Y is expected to act locally to kill the tumour cells.

What is the stage of development of this medicine?

The effects of yttrium (⁹⁰Y) edotreotide have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with gastro-entero-pancreatic neuroendocrine tumours were ongoing.

At the time of submission, yttrium (⁹⁰Y) edotreotide was not authorised anywhere in the world for gastro-entero-pancreatic neuroendocrine tumours. Orphan designation of yttrium (90Y) edotreotide had been granted in the United States for the treatment of somatostatin-positive gastro-entero-pancreatic neuroendocrine tumours.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 October 2008 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 five in 10,000 people in the Community) 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.

EU/3/08/589: Public summary of positive opinion for orphan designation of yttrium (90Y) edotreotide for the treatment of gastro-entero-pancreatic neuroendocrine tumours

Adopted Reference Number: EMEA/COMP/547022/2008 Rev.2

Key facts

• Active substance:
  yttrium (90Y) edotreotide
• Intended use:
  Treatment of gastro-entero-pancreatic neuroendocrine tumours
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/08/589
• Date of designation:
  04/08/2008
• Sponsor:

  Molecular Insight Limited; Eversheds House; 70 Great Bridgewater Street; Manchester M1 5ES; United Kingdom; Tel. [phone]; Fax: [phone]