EU/3/01/081

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Orphan designation

On 4 February 2002, orphan designation (EU/3/01/081) was granted by the European Commission to Sanofi-Synthélabo, France, for fumagillin for the treatment of diarrhoea associated with intestinal microsporidial infection.

In May 2006, Sanofi-Synthélabo changed name to Sanofi Aventis and in October 2012 Sanofi Aventis changed name to Sanofi-Aventis Groupe.

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

What is intestinal microsporidial infection?

Microsporidia are intestinal parasites that can cause severe chronic diarrhoea and malabsorption especially in patients with immunodeficiency such as patients with AIDS and in patients undergoing transplantation. The infection is life-threatening.

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

At the time of designation, diarrhoea associated with intestinal microsporidial infection affected approximately 1.3 to 2.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 49,000 to 91,000 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).


*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. At the time of designation, this represented a population of 380,600,000 (Eurostat 2002).

What treatments are available?

No satisfactory treatment existed that had been authorised at the time of designation, although different antiparasitic drugs had shown symptomatic improvement but without eradicating the microsporidia from the intestinal tract.

How is this medicine expected to work?

The mechanism of action is not fully known but fumagillin is thought to inhibit the RNA synthesis and growth of microsporidial parasites.

What is the stage of development of this medicine?

The effects of fumagillin have been evaluated in experimental models, and clinical trials for the treatment of diarrhoea associated with intestinal microsporidial infection have been performed. Fumagillin is currently being developed for treatment of diarrhoea associated with intestinal microsporidial infection in severely immunosuppressed AIDS patients.

The product was not authorised or designated as orphan medicinal product in any country at the time of submission of the application.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 November 2001 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.

Key facts

Product details for <p>Fumagillin</p>
Active substanceFumagillin
Medicine Name
Disease/conditionTreatment of diarrhoea associated with intestinal microsporidial infection
Date of decision04/02/2002
OutcomePositive
Orphan decision numberEU/3/01/081

Review of designation

Sponsor’s contact details

Sanofi-Aventis Groupe
54 rue de la Boétie
75008 Paris
France
Tel. +33 1 53 77 40 00
Fax +33 1 53 77 41 33
www.sanofi-aventis.com/contact/contact.asp

Patients’ organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.