EU/3/13/1149 - orphan designation for prevention of ischaemia/reperfusion injury associated with solid organ transplantation

human haemin

Overview

On 17 July 2013, orphan designation (EU/3/13/1149) was granted by the European Commission to Borders Technology Management Ltd, United Kingdom, for human hemin for the prevention of ischaemia reperfusion injury associated with solid-organ transplantation.

What is ischaemia reperfusion injury associated with solid-organ transplantation?

Ischaemia reperfusion injury is tissue damage that can occur in transplant organs as a result of the period between donation and transplant when they are kept outside the body with no blood supply. The lack of blood supply, if prolonged, can cause damage to the organ known as ischaemia. When the organ is transplanted into a recipient, the return of blood supply (reperfusion) then causes inflammation and further damage to the organ. These processes increase the risk of the organ not working or being rejected by the recipient.

Because ischaemia reperfusion injury associated with solid-organ transplantation impairs the functioning of the organ, it is a life-threatening condition for the recipient.

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 ischaemia reperfusion injury associated with solid-organ transplantation was estimated to be approximately 0.6 people in 10,000 per year in the European Union (EU). This was equivalent to a total of around 31,000 people per year*, and 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).

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*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 509,000,000 (Eurostat 2013).

What methods of prevention are available?

At the time of designation methods to prevent ischaemia reperfusion injury associated with solid-organ transplantation included the storage of the organ in cold conditions in special preservation solutions. Two such solutions were authorised for organ preservation in some countries of the EU at the time of designation.

The sponsor has provided sufficient information to show that human haemin might be of significant benefit for patients at risk of developing ischaemia reperfusion injury because it works in a different way to existing methods and can be combined with them; early studies show that it might improve the outcome of transplantation. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Human haemin is a substance naturally found in red blood cells. It increases the production of an enzyme called haeme oxygenase-1 that can help protect cells against inflammation and cell death. When given to patients who are receiving an organ transplant, these effects are expected to reduce the damage caused by ischaemia reperfusion injury and so improve the functioning of the transplanted organ.

What is the stage of development of this medicine?

The effects of human hemin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, a clinical trial with human hemin in patients at risk of ischaemia reperfusion injury associated with solid-organ transplantation was ongoing.

At the time of submission, human haemin was authorised in several EU Member States for treatment of acute attacks of hepatic porphyria. It was not authorised anywhere in the EU for prevention of ischaemia reperfusion injury associated with solid-organ transplantation or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 June 2013 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/3/13/1149: Public summary of opinion on orphan designation: Human haemin for the prevention of ischaemia reperfusion injury associated with solid-organ transplantation

Adopted Reference Number: EMA/COMP/399770/2013

Key facts

• Active substance:
  human haemin
• Intended use:
  Prevention of ischaemia/reperfusion injury associated with solid organ transplantation
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/13/1149
• Date of designation:
  17/07/2013
• Sponsor:

  Borders Technology Management Ltd; 2 Broomlee Mains Court; West Linton; Peeblesshire; EH46 7BP; Scotland; United Kingdom; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.