EU/3/09/638

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Orphan designation

On 15 May 2009, orphan designation (EU/3/09/638) was granted by the European Commission to Opsona Therapeutics, Ireland, for humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 for the prevention of ischaemia / reperfusion injury in solid-organ transplantation.

What is ischaemia / reperfusion injury in solid-organ transplantation?

Ischaemia and reperfusion injury are problems that can occur during an organ transplant. In the course of a transplant, the organ to be transplanted (the ‘graft’) needs to survive outside the body with no blood supply for a short while. This decrease in blood supply is called ischaemia, and, if prolonged, can cause damage to the organ because of the lack of oxygen and nutrients. When the graft is attached to the recipient’s blood circulation, the restoration of blood supply to the organ (reperfusion) can cause inflammation and damage to the organ (reperfusion injury). These processes increase the risk of the graft not working or being rejected by the recipient.

Because ischaemia / reperfusion injury in solid-organ transplantation is a threat to the correct function of the graft, it is a life-threatening condition for the recipient of the graft.

What is the estimated number of patients affected by the condition?

At the time of designation, the number of patients at risk of ischaemia / reperfusion injury in solid-organ transplantation was estimated to be less than 1 people in 10,000 in the European Union (EU). This was equivalent to a total of fewer than 50,000 people*, 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).


*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. At the time of designation, this represented a population of 504,800,000 (Eurostat 2009).

What methods of prevention are available?

To prevent problems during organ transplant, the grafts are usually stored in cold conditions and in solutions that reduce the effects of ischaemia. The sponsor has provided sufficient information to show that humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 might be of significant benefit for patients at risk of ischaemia / reperfusion injury in solid-organ transplantation because it may work in a way that could limit the damage to grafts after they have been transplanted. 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?

Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure (called an antigen). This medicine attaches to toll-like receptor type 2 (TLR2), a protein in the immune system (the body’s natural defences) that plays a role in inflammation and damage caused by ischaemia and reperfusion.

The medicine is to be given to the patient by injection into a vein, just before the graft is transplanted. After injection, the antibody is expected to attach to TLR2 in the blood. This may limit the activation of TLR2 once the organ is grafted, and decrease inflammation, reducing the damage to the graft.

What is the stage of development of this medicine?

The effects of this medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients at risk of ischaemia / reperfusion injury in solid-organ transplantation had been started.

At the time of submission, this medicine was not authorised anywhere in the EU for the prevention of ischaemia / reperfusion injury in solid-organ transplantation or designated as 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 2 April 2009 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 Community) 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.

Key facts

Product details for <p>Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2</p>
Active substanceHumanised IgG4 monoclonal antibody to the human toll-like receptor type 2
Medicine Name
Disease/conditionPrevention of the ischaemia / reperfusion injury associated with solid-organ transplantation
Date of decision15/05/2009
OutcomePositive
Orphan decision numberEU/3/09/638

Review of designation

Sponsor’s contact details:

Opsona Therapeutics
2nd Floor
Ashford House
Tara Street
Dublin 2
Ireland
Tel. +353 1 677 0223
Fax +353 1 677 8949
E-mail: info@opsona.com

Patients' organisations:

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.