EU/3/06/426 - orphan designation for treatment of primary malignant bone tumours

Fenretinide

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in February 2011 on request of the sponsor.

On 26 January 2007, orphan designation (EU/3/06/426) was granted by the European Commission to Cancer Research UK, United Kingdom, for fenretinide for the treatment of primary malignant bone tumours.

What are primary malignant bone tumours?

Tumours that arise in the bone tissues are known as primary malignant bone tumours. Primary malignant bone tumours are classified by the type of tissue they originate, the most common being bone sarcomas. They can occur in any bone of the body and they spread almost exclusively through the blood. There are many types of primary malignant bone tumours, which tend to behave differently. Having a primary malignant bone tumour is a serious condition, potentially debilitating and life-threatening.

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

At the time of designation, primary malignant bone tumours affected less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 46,000 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).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What treatments are available?

Surgery is currently the main choice of therapy for primary malignant bone tumours. It is usually combined with chemotherapy (using drugs to kill cancer cells) prior to or after surgery. Several products were authorised for the condition in some Member States in the Community at the time of submission of the application for orphan drug designation.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that fenretinide might be of potential significant benefit for the treatment of primary malignant bone tumours mainly because it might improve the long-term outcome of the patients. This assumption 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?

Fenretinide is structurally related to vitamin A. The exact mechanism of action of fenretidine is not fully understood but it is thought that it binds to, and kills tumour cells in primary malignant bone tumours.

What is the stage of development of this medicine?

The effects of fenretinide were evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with primary malignant bone tumours were initiated. However some clinical trials were planned, at the time of submission.

Fenretinide was not authorised anywhere worldwide for treatment of primary malignant bone tumours or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 December 2006 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/3/06/426: Public summary of positive opinion for orphan designation of fenretinide for the treatment of primary malignant bone tumours

Adopted Reference Number: EMEA/COMP/96682/2008 Rev. 1

Key facts

• Active substance:
  Fenretinide
• Intended use:
  Treatment of primary malignant bone tumours
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/06/426
• Date of designation:
  26/01/2007
• Sponsor:

  Cancer Research UK; 61 Lincoln's Inn Fields; London, WC2A 3PX; United Kingdom; Telephone: [phone]; Telefax: 44 207 121 69 02; [e-mail]