Overview

On 19 February 2014, orphan designation (EU/3/14/1238) was granted by the European Commission to Alexion Europe SAS, France, for eculizumab for the prevention of delayed graft function after solid organ transplantation.

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in April 2017 on request of the Sponsor.

Delayed graft function is the failure of a transplanted organ to start working properly in the first few days after the transplant. Delayed graft function following transplantation of a solid organ can occur as a result of damage to the organ caused by the interruption and restoration of blood flow. This is called 'ischaemia/reperfusion injury' and is associated with an inflammatory reaction, caused in part by the invasion of neutrophils (a type of white blood cell) into the transplanted organ.

Delayed graft function after solid organ transplantation is a debilitating and life-threatening condition because of the risk of losing the transplanted organ.

At the time of designation, the number of patients at risk of delayed graft function after solid organ transplantation was estimated to be not more than 0.6 people in 10,000 in the European Union (EU). This was equivalent to a total of not more than 31,000 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 28), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 512,900,000 (Eurostat 2014).

At the time of designation, no satisfactory methods were authorised in the EU for the prevention of delayed graft function after solid organ transplantation. Several preventative measures were commonly used to reduce the risk of delayed graft function, including careful selection of the organ donor and preservation of the organ during transport.

Eculizumab is a monoclonal antibody, a type of protein that has been designed to recognise and attach to a specific structure (called an antigen) that is found in the body. It is expected to work by attaching and blocking the action of a protein called the C5 complement protein, which is involved in the inflammatory processes that can cause injury to the transplanted organ. This is expected to reduce the risk of delayed graft function after solid organ transplantation.

The effects of eculizumab have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with eculizumab in patients who had undergone solid organ transplantation were ongoing.

At the time of submission, eculizumab was not authorised anywhere in the EU for preventing delayed function after solid organ transplantation or designated as an orphan medicinal product elsewhere for this indication.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 January 2014 recommending the granting of this designation.

  • 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/3/14/1238: Public summary of opinion on orphan designation: Eculizumab for the prevention of delayed graft function after solid organ transplantation

AdoptedReference Number: EMA/COMP/36006/2014 Rev. 1

English (EN) (135.25 KB - PDF)

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Key facts

Active substance
Eculizumab
Intended use
Prevention of delayed graft function after solid organ transplantation
Orphan designation status
Withdrawn
EU designation number
EU/3/14/1238
Date of designation
Sponsor

Alexion Europe S.A.S.

Review of designation

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

EMA list of opinions on orphan medicinal product designation

EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Patients' organisations

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

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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