EU/3/06/427 - orphan designation for treatment of soft tissue sarcoma

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Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in February 2011 on request of the sponsor.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 30 January 2007, orphan designation (EU/3/06/427) was granted by the European Commission to Cancer Research UK, United Kingdom, for fenretinide for the treatment of soft tissue sarcoma.

What is soft tissue sarcoma?

Soft tissue sarcomas are cancers of the supporting tissues of the body. They can occur in muscle, fat, blood vessels or in any of the other tissues that support, surround and protect the organs of the body. There are many types of soft tissue sarcoma. The cause of soft tissue sarcoma is largely unknown. Soft tissue sarcoma 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, soft tissue sarcoma affected less than 2.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 115,000 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 early stage soft tissue sarcomas. For larger sarcomas, and where it is thought there is a possibility of cancer cells being left behind, after a surgical intervention, radiotherapy (using high-dose x-rays or other high-energy rays to kill cancer cells) and chemotherapy (using drugs to kill cancer cells) are usually used. Several products for treatment of soft tissue sarcoma 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 soft tissue sarcoma 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 soft tissue sarcomas.

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 soft tissue sarcoma were initiated.

Fenretinide was not authorised anywhere worldwide for treatment of soft tissue sarcoma 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/3/06/427: Public summary of positive opinion for orphan designation of fenretinide for the treatment of soft tissue sarcoma

Adopted Reference Number: EMA/COMP/96616/2008 Rev.1

Key facts

• Active substance:
  Fenretinide
• Intended use:
  Treatment of soft tissue sarcoma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/06/427
• Date of designation:
  30/01/2007
• Sponsor:

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