EU/3/04/187 - orphan designation for prevention of hepatitis-B re-infection following liver transplantation

human monoclonal hepatitis B immunoglobulins

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in July 2010 on request of the sponsor.

On 23 February 2004, orphan designation (EU/3/04/187) was granted by the European Commission to ICON Clinical Research (UK) Ltd, United Kingdom, for human monoclonal hepatitis B immunoglobulins for the prevention of hepatitis B re-infection following liver transplantation.

What is hepatitis B re-infection following liver transplantation?

Hepatitis is a general term that means inflammation of the liver. Hepatitis B is an infectious disease that affects the liver, caused by the hepatitis B virus. Symptoms may not be seen for 6 weeks to 6 months after infection. Sometimes people who have been infected do not look or feel ill. Others have mild flu-like symptoms, but some people can become very ill.

Most adults fully recover from the disease. But some people, particularly children, who may not have had symptoms, can remain infected. They are called hepatitis B virus carriers. Hepatitis B 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 in body fluids (such as blood) of hepatitis B virus carriers, the transplanted liver might become infected on its turn.

Hepatitis B re-infection following liver transplantation is chronically debilitating and life-threatening.

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

At the time of designation, the number of patients at risk of hepatitis B re-infection following liver transplantation was estimated to be approximately 0.2 people in 10,000 in the European Union (EU)*. This is equivalent to a total of around 7,700 people, which is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 385,000,000 (Eurostat 2002) and may differ from the true number of patients

What methods of prevention are available?

Immunoglobulins were authorised in the Community for the prevention of hepatitis B re-infection following liver transplantation at the time of submission of the application for orphan drug designation, whilst other medicinal products with anti-viral activity were widely used.

Human monoclonal hepatitis B immunoglobulins might be of potential significant benefit for prevention of the condition especially since preliminary results have shown a high specific activity against the virus. These benefits will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Antibodies are proteins, which specifically recognise and attach themselves to certain foreign substances, such as proteins found on the surface of viruses or bacteria. The term “monoclonal” means that the antibody is produced using cells that have identical genes, so each cell producing exactly the same antibody molecules. Human monoclonal hepatitis B immunoglobulins are antibodies that neutralise hepatitis B virus. This would prevent that the liver transplant becomes infected.

What is the stage of development of this medicine?

The effects of human monoclonal hepatitis B immunoglobulins were evaluated in experimental models. At the time of submission of the application for orphan designation, no clinical trials in patients who received liver transplantation due to chronic hepatitis B liver disease were initiated.

Human monoclonal hepatitis B immunoglobulins were not marketed anywhere worldwide for prevention of hepatitis B re-infection following liver transplantation or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 14 January 2004 recommending the granting of this 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;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or 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 quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/04/187: Public summary of positive opinion for orphan designation of human monoclonal hepatitis B immunoglobulins for the prevention of hepatitis B re-infection following liver transplantation

Adopted Reference Number: EMA/COMP/29/2004 Rev.1

Key facts

• Active substance:
  human monoclonal hepatitis B immunoglobulins
• Intended use:
  Prevention of hepatitis-B re-infection following liver transplantation
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/04/187
• Date of designation:
  23/02/2004
• Sponsor:

  ICON Consulting; ICON Clinical Research (UK) Ltd.; 2 Globeside, Globeside Business Park; Marlow, Bucks SL7 1TB; United Kingdom; Telephone: [phone]; Telefax: (44-1628) 49 63 01; E-mail: [e-mail]