EU/3/06/408

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Orphan designation

On 31 October 2006, orphan designation (EU/3/06/408) was granted by the European Commission to Biotest Pharma GmbH, Germany, for human cytomegalovirus immunoglobulin for the prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection in pregnant women.

What is congenital cytomegalovirus infection following primary cytomegalovirus infection?

Cytomegalovirus is a virus that infects many people worldwide. In the majority of cases the virus is latent (inactive) and causes neither disease nor any symptoms of any kind in man. Usually, the virus is only activated in patients who, for some reason, have a weaker immune system. Cytomegalovirus disease can affect several organs in the body, such as the eyes, liver, lungs and the gastrointestinal tract (the stomach and intestines) and cause organ failure. If a woman is infected (primary infection) with cytomegalovirus while pregnant, the virus can pass to the unborn baby who does not yet have a very strong immune system. When congenital (in-born), the disease can also cause growth retardation and brain damage. Congenital cytomegalovirus disease is chronically debilitating and life-threatening.

What is the estimated number of patients at risk of developing the condition?

At the time of designation cytomegalovirus infection affected approximately 1.4 in 10,000 people in the European Union (EU) *. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 65,000 people.

*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).

What methods of prevention are available?

There were no medicinal products authorised for the prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection in pregnant women in the Community at the time of submission of application for orphan drug designation.

How is this medicine expected to work?

Human cytomegalovirus immunoglobulin is a mix of antibodies (proteins of the immune system that can bind to a specific molecular structure) directed at cytomegalovirus particles. Antibodies can neutralise infectious agents or start processes that lead to their destruction by cells of the immune system. According to the sponsor, human cytomegalovirus immunoglobulin will thus reduce the amount of cytomegalovirus in the pregnant woman’s blood and reduce the risk of the unborn baby becoming infected.

What is the stage of development of this medicine?

The effects of human cytomegalovirus immunoglobulin were evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in pregnant women with primary cytomegalovirus infection were ongoing.
The medicinal product was not designated as orphan medicinal product anywhere worldwide, 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 6 September 2006 a positive opinion recommending the grant of the above-mentioned 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Human cytomegalovirus immunoglobulin</p>
Active substanceHuman cytomegalovirus immunoglobulin
Medicine Name
Disease/conditionPrevention of congenital cytomegalovirus infection following primary cytomegalovirus infection
Date of decision30/10/2006
OutcomePositive
Orphan decision numberEU/3/06/408

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