EU/3/07/488 - orphan designation for treatment of gastro-entero-pancreatic neuroendocrine tumours

everolimus

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in July 2011 on request of the sponsor.

On 14 November 2007, orphan designation (EU/3/07/488) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for everolimus for the treatment gastro-entero-pancreatic neuroendocrine tumours.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What are gastro-entero-pancreatic neuroendocrine tumours?

Gastro-entero-pancreatic neuroendocrine tumours (GEP-NET) are a group of tumours that share a number of common characteristics, for example they contain specific chemical substances called “neuroendocrine markers” on the tumour cell surface and they are able to produce hormones. There are two main types of GEP-NET: carcinoid tumours, and “pancreatic-type” endocrine tumours (these often occur in the pancreas, but also in other sites). GEP-NET are chronically debilitating as they often produce and secrete hormonal substances that may cause severe symptoms, and 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-NET affected approximately 1.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 90,000 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).

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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 500,300,000 (Eurostat 2007).

What treatments are available?

At the time of the application for orphan designation, several products were authorised in the Community for the treatment of one or more of GEP-NET. In most cases, these products are active against the symptoms caused by the secretion of hormones, but are not active against the growth of the tumours.

Everolimus may be of potential significant benefit, as it is expected to be active against tumour growth, and thus reduce tumour size and improve survival. These assumptions will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Enzymes are proteins produced by the human body that speed up the conversion of certain chemical substances of the body into other substances. Everolimus blocks (inhibits) a particular enzyme, called serine/threonine kinase. This enzyme plays a role in a number of molecular reactions, which control the growth and the division of the cells. In cancer cells, the function of this enzyme is disturbed, causing uncontrolled growth and multiplication of the cancer cells. Everolimus might, by inhibition of this enzyme activity, help slow down or stop the further growth of the cancer cells.

What is the stage of development of this medicine?

At the time of orphan designation the effects of everolimus were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with GEP-NET were ongoing.

Everolimus was not authorised anywhere worldwide for treatment of GEPNET, at the time of submission. Orphan designation of everolimus was granted in the European Union for the treatment of renal cell carcinoma in 2007.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 September 2007 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.

EU/3/07/488: Public summary of positive opinion for orphan designation of everolimus for the treatment of gastro-entero-pancreatic neuroendocrine tumours

Adopted Reference Number: EMEA/COMP/467747/2007 Rev. 2

Key facts

• Active substance:
  everolimus
• Intended use:
  Treatment of gastro-entero-pancreatic neuroendocrine tumours
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/07/488
• Date of designation:
  14/11/2007
• Sponsor:

  Novartis Europharm Limited