EU/3/05/319

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Orphan designation

On 3 November 2005, orphan designation (EU/3/05/319) was granted by the European Commission to Nabi Biopharmaceuticals Europe Ltd, Ireland, for human Staphylococcus aureus immunoglobulin for the treatment of Staphylococcus aureus bacteraemia.
The sponsorship was transferred to Biotest Pharma GmbH, Germany, in February 2008.

What is Staphylococcus aureus bacteraemia?

Staphylococcus aureus is a microorganism that is a normal component of the human flora, which are the microorganisms that normally live in areas of our body. However sometimes these microorganisms can cause infections in human beings, particularly in patients with a compromised defence system (immunity). Staphylococcus aureus can have a harming effect directly through the effect of a toxic substance (toxin) that it is able to produce. Staphylococcus aureus can cause a range of different diseases, from mild skin infections to life-threatening generalised (systemic) infections.
Bacteraemia is defined as the presence of a microorganism in the blood. This way microorganisms spread from a localised point of infection to a systemic infection and affect organs that are far from the original site of the infection.
Staphylococcus aureus bacteraemia is life threatening, as it can lead to the development of severe complications.

What are the methods of treatment available?

Staphylococcus aureus bacteraemia should be treated with appropriate antibiotics. Antibiotics are drugs that are able to kill microorganisms. The choice of antibiotic is usually dictated in the clinical practice by the type of the microorganism causing the infection and a number of factors like the primary localisation of the infection in the body, the immune situation of the patient, the risk for complications, the presence of other diseases, etc. Several antibiotics were authorised for the condition in some countries in the Community at the time of submission of the application for orphan drug designation.
Staphylococcus aureus immunoglobulin might be of potential significant benefit for patients affected by the condition because it would offer an alternative treatment that could be used in combination with antibiotics. 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, Staphylococcus aureus bacteraemia was considered to affect approximately 2.4 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 110,000 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?

Immunoglobulins are proteins that the human immune system produces to fight infection or any external intruder in the body. The human Staphylococcus aureus immunoglobulin was obtained by giving a vaccine containing Staphylococcus aureus to healthy people who then produced the natural immunoglobulin against that microorganism. The human Staphylococcus aureus immunoglobulin is then purified from the plasma of the vaccinated persons and processed into the final medicinal product form. This product, administered to a patient with Staphylococcus aureus in his blood would recognise the micro-organism and would interact with it helping other cells of the patient to kill the micro-organism.

What is the stage of development of this medicinal product?

At the time of submission of the application for orphan designation, clinical trials in patients with Staphylococcus aureus bacteraemia were ongoing.
Human Staphylococcus aureus immunoglobulin was not authorised anywhere worldwide for Staphylococcus aureus bacteraemia, at the time of submission.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 9 September 2005 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>Human Staphylococcus aureus immunoglobulin</p>
Active substanceHuman Staphylococcus aureus immunoglobulin
Medicine Name
Disease/conditionTreatment of Staphylococcus aureus bacteraemia
Date of decision02/11/2005
OutcomePositive
Orphan decision numberEU/3/05/319

Review of designation

Sponsor’s contact details:

Biotest Pharma GmbH
Landsteinerstr. 5
63303 Dreieich
Germany
Telephone: + 49 6103 801 588
Telefax: + 49 6103 801 180
E-mail: mail@biotest.de

Patients’ association contact point:

Not available