EU/3/07/430

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Orphan designation

On 20 February 2007, orphan designation (EU/3/07/430) was granted by the European Commission to ACE Pharmaceuticals BV, The Netherlands, for artesunate for the treatment of malaria.

What is malaria?

Malaria is an infectious disease caused by Plasmodium parasites. There are four species of Plasmodium that may infect humans: P vivax, P. malariae, P. ovale and P. falciparum. Malaria is carried from person to person by infected Anopheles mosquitoes, and transmission occurs mostly in tropical and subtropical regions including parts of the Americas, Asia, and Africa. In Europe, malaria is mainly encountered in travellers returning from these areas. Following a bite from an infected mosquito, the parasite goes to the liver. When the parasite then enters the bloodstream it infects and destroys red blood cells.

Fever is the major symptom of malaria. The disease must always be suspected when fever occurs during or after travel to an infected area. In addition, flu-like symptoms such as chills, headaches, muscle-aches, and fatigue can occur. Malaria due to P. falciparum may progress rapidly causing seizures, coma, breathing and kidney problems. The disease is life-threatening, and active malaria infection with P. falciparum is a medical emergency.

What are the methods of treatment available?

Several medicinal products with anti-malarial activity were authorised for the condition 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 artesunate might be of potential significant benefit for the treatment of malaria, mainly because it might improve survival in those suffering severe, life-threatening malaria. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

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

Based on the information provided by the sponsor and previous knowledge of the Committee, malaria was considered to affect approximately 0.3 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 15,000 persons.

*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.

How is this medicinal product expected to act?

Artesunate is a chemically modified derivative of the natural product artemisinin, which is found in sweet wormwood. It is proposed that it acts by damaging the membrane of the parasite, thus killing the parasite.

What is the stage of development of this medicinal product?

The effects of artesunate have been evaluated in experimental models.

At the time of submission of the application for orphan designation, artesunate was not authorised in the European Union.

Orphan designation of intravenously administered artesunate had been granted in the United States for immediate treatment of malaria.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 10 January 2007 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition,
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.

Key facts

Product details for <p>Artesunate</p>
Active substanceArtesunate
Medicine Name
Disease/conditionTreatment of malaria
Date of decision19/02/2007
OutcomePositive
Orphan decision numberEU/3/07/430

Review of designation

Sponsor’s contact details:

ACE Pharmaceuticals BV
Schepenveld 41, 3891 ZK Zeewolde
The Netherlands
Telephone: +31 36 522 72 01
Telefax: +31 36 522 9096
E-mail: rvdv@ace-pharm.nl

Patients’ associations contact points:

Not available