EU/3/04/246 - orphan designation for treatment of familial adenomatous polyposis (FAP)

valproic acid

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in November 2012 on request of the sponsor.

On 30 November 2004, orphan designation (EU/3/04/246) was granted by the European Commission to G2M Cancer Drugs AG, Germany for valproic acid, sodium for the treatment of familial adenomatous polyposis.

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

What is familial adenomatous polyposis?

Familial adenomatous polyposis (FAP), also known as familial polyposis coli, is an inherited disease characterised by the appearance of numerous masses called 'polyps' throughout the large bowel. Other abnormalities involving other organs such as the eyes, the bones or the skin can also be present. The disease is caused by a defect (called mutation) in the gene that normally inhibits (blocks) the formation of the polyps. Patients with FAP usually have to undergo removal of a big part of their bowel, which is chronically debilitating. FAP may also lead to cancer of the large bowel and as such is a life-threatening condition.

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

At the time of designation, familial adenomatous polyposis affected approximately 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 23,000 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 464,200,000 (Eurostat 2004).

What are the methods of treatment available?

Available non-pharmacological methods for the treatment of FAP consist of surgical removal of the large bowel followed by regular visual inspections of the bowel cavities using a flexible instrument called an endoscope. Medicinal treatments such as anti-inflammatory agents were authorised and used for the condition in the Community at the time of submission of the application for orphan designation.

Valproic acid, sodium might be of potential significant benefit for the treatment of familial adenomatous polyposis because it may act in a different way than other available treatments. 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?

Valproic acid, sodium is expected to stop (inhibit) the formation of the new polyps and it might also reduce the size of the existing polyps. Although it is not yet completely understood how it works, it is suggested by the sponsor that it might act on a certain substance normally produced by the body cells, the so-called histone deacetylase, that is involved in the generation of the polyps.

What is the stage of development of this medicine?

The effects of valproic acid, sodium have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with familial adenomatous polyposis had been initiated.

Valproic acid, sodium was not marketed anywhere worldwide for the treatment of familial adenomatous polyposis or designated as an 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 7 October 2004 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/3/04/246: Public summary of positive opinion for orphan designation of valproic acid, sodium for the treatment of familial adenomatous polyposis

Adopted Reference Number: EMEA/COMP/109745/2004, Rev. 2

Key facts

• Active substance:
  valproic acid
• Intended use:
  Treatment of familial adenomatous polyposis (FAP)
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/04/246
• Date of designation:
  30/11/2004
• Sponsor:

  G2M Cancer Drugs AG; Paul-Ehrlich Str. 42-44; D-60590 Frankfurt; Germany; Tel. [phone]; Fax [phone]; E-mail: [e-mail]