EU/3/05/295

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Orphan designation

On 8 July 2005 orphan designation (EU/3/05/295) was granted by the European Commission to Nabi Biopharmaceuticals Europe Ltd, Ireland, for hepatitis C immunoglobulin for the prevention of recurrent hepatitis C virus induced liver disease in liver transplant recipients.
The sponsorship was transferred to Biotest Pharma GmbH, Germany, in February 2008.

What is recurrent hepatitis C virus induced liver disease in liver transplant recipients?

Hepatitis is a general term that means inflammation of the liver. Hepatitis C is an infectious disease that affects the liver, caused by the hepatitis C virus. This viral infection does not always manifest itself clinically, and thus some people infected with the virus will not look or feel ill, others might have mild flu-like symptoms, or can become very ill.
Most patients fully recover spontaneously while others remain infected, initially without any sign or symptom of the disease. They are called hepatitis C virus carriers. Hepatitis C carriers are at risk of serious liver disease, such as cirrhosis (liver scarring) and liver cancer. In these cases of advanced liver disease, liver transplantation (getting a liver from another person) could become a necessity. However, since the virus is found also in body fluids (such as blood) of hepatitis C virus carriers, the transplanted liver might become infected on its turn and the liver disease can start in the transplanted liver.
Recurrent hepatitis C virus induced liver disease in liver transplant recipients is chronically debilitating and life-threatening.

What are the methods of prevention available?

No satisfactory methods exist that were authorised at the time of application.

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

Based on the information provided by the sponsor and previous knowledge of the Committee, the number of patients at risk of recurrent hepatitis C virus induced liver disease in liver transplant recipients was considered to be less than 1 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 46,000 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?

Antibodies are proteins, which specifically recognise and attach themselves to certain structures, such as proteins found on the surface of viruses or bacteria. Hepatitis C immunoglobulin is an antibody that recognises, attaches and neutralises the hepatitis C virus. In experimental models it has been shown that hepatitis C immunoglobulin would prevent recurrent hepatitis C virus induced liver disease. Therefore, if administered after liver transplantation, it would protect the new liver from the harmful activity of the hepatitis C virus.

What is the stage of development of this medicinal product?

The effects of hepatitis C immunoglobulin were evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in patients who received liver transplantation due to chronic hepatitis C liver disease were initiated.
Hepatitis C immunoglobulin was not authorised anywhere worldwide for recurrent hepatitis C virus induced liver disease in liver transplant recipients, at the time of submission. Orphan designation of hepatitis C virus immune globulin (human) was granted in the United States for the condition of prophylaxis of hepatitis C infection in liver transplant recipients.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 12 May 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>Hepatitis C immunoglobulin</p>
Active substanceHepatitis C immunoglobulin
Medicine Name
Disease/conditionPrevention of recurrent hepatitis C virus induced liver disease in liver transplant recipients
Date of decision08/07/2005
OutcomePositive
Orphan decision numberEU/3/05/295

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’ associations contact points:

Euroliver Foundation
Markgravestraat, 10 P.O.
B-2000 Anvers
Belgium
Telephone: +32 3 226 79 97
Telefax: +32 3 226 79 70
E-mail: euroliver@glo.be

British Liver Trust
Portman House
44 High Street
Ringwood
BH24 1AG
United Kingdom
Telephone: +44 14 25 46 30 80
Telefax: +44 14 25 47 07 06