EU/3/07/503

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Orphan designation

On 6 December 2007, orphan designation (EU/3/07/503) was granted by the European Commission to GENimmune, N.V., Belgium, for recombinant human hepatitis C monoclonal antibody against C4 region of E1 for the prevention of recurrent hepatitis C virus induced liver disease in liver transplant recipients.

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 cause symptoms in the patient, 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 and they are at risk of serious liver disease, such as cirrhosis (liver scarring) and liver cancer. In these cases, liver transplantation (getting a liver from a donor) could become a necessity. However, since the virus is found also in other organs of the body of hepatitis C virus carriers, the transplanted liver might become infected on its turn and the liver disease could start again 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 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, recurrent hepatitis C virus induced liver disease in liver transplant recipients was considered to affect approximately 0.1 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 5000 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 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). 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. Recombinant human hepatitis C monoclonal antibody against C4 region of E1 is an antibody that recognises and neutralises (prevents from being harmful) a specific structure found on hepatitis C virus particles. The sponsor has suggested that recombinant human hepatitis C monoclonal antibody against C4 region of E1 would prevent recurrent hepatitis C virus induced liver disease through this mechanism. Thus, if administered after liver transplantation, it could potentially protect the new liver from becoming infected by hepatitis C virus.

What is the stage of development of this medicinal product?

The evaluation of the effects of recombinant human hepatitis C monoclonal antibody against C4 region of E1 in experimental models was ongoing. At the time of submission of the application for orphan designation, no clinical trials in patients with recurrent hepatitis C virus induced liver disease in liver transplant recipients were initiated.

Recombinant human hepatitis C monoclonal antibody against C4 region of E1 was not authorised anywhere worldwide for the prevention of recurrent hepatitis C virus induced liver disease in liver transplant recipients or designated as orphan medicinal product elsewhere for this condition, 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 10 October 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>Recombinant human hepatitis C monoclonal antibody against C4 region of E1</p>
Active substanceRecombinant human hepatitis C monoclonal antibody against C4 region of E1
Medicine Name
Disease/conditionPrevention of recurrent hepatitis C virus induced liver disease in liver transplant recipients
Date of decision05/12/2007
OutcomePositive
Orphan decision numberEU/3/07/503

Review of designation

Sponsor’s contact details:

GENimmune, N.V.
Technologiepark 6
9052 Zwijnaarde
Belgium
Telephone: + 32 9 2410 737
Telefax: + 32 9 329 2410 907
E-mail: contact@genimmune.be

Patients’ associations contact points:

British Liver Trust
Portman House
44 High Street
Ringwood
BH24 1AG
United Kingdom
Telephone: +44 14 25 46 30 80
E-mail: info@britishlivertrust.org.uk

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