EU/3/06/376

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Orphan designation

On 22 May 2006 orphan designation (EU/3/06/376) was granted by the European Commission to RJM Consultancy Ltd, United Kingdom, for recombinant P-selectin glycoprotein immunoglobulin for the prevention of post transplantation graft dysfunction.

What is post transplantation graft dysfunction?

Organ transplant refers to the transfer of organs from one body to another. Once the organ is transplanted, it is called a graft. The transplant requires complex surgical procedures. Organs always suffer during this process, due to different causes. In some cases, the damage is such that the graft function becomes abnormal, which in case of kidney transplant results in a range of alterations that go from a delayed normalisation of laboratory parameters to the inability of the transplanted kidney to perform its normal function. This condition is called post transplantation graft dysfunction.

The dysfunction depends on the type of injury. For example, the disruption of blood supply may cause an immediate damage to the organ due to the lack of oxygen. In other cases, the patient has an immune reaction against the graft he received (in this case the immune system, the body’s natural defence mechanism, attacks and aims to destroy the new organ/graft). In the long run, the dysfunction can cause graft failure and rejection. Post transplantation graft dysfunction is a long term debilitating and life-threatening condition.

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 post transplantation graft dysfunction was estimated to be approximately 0.5 people in 10,000 in the European Union (EU)*. This is equivalent to a total of around 23,000 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: 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 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What methods of prevention are available?

There were several medicines that were authorised to prevent graft dysfunction, and organ rejection in the Community at the time of submission of the application for orphan drug designation. These medicines consisted of chemicals and antibodies (proteins of the immune system that can recognise and bind to specific structures) that inhibit the immune response against the graft. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that recombinant P-selectin glycoprotein immunoglobulin might be of potential significant benefit because it might act differently from available treatments. The assumption 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?

The situation where cells do not receive their normal supply of oxygen and nutrients is called ischemia. Organs that are extracted for transplantation suffer ischemia to a certain extent, due to the normal process of extraction and transport. In this situation cells react by placing certain proteins on the cell surface. These proteins can attract white blood cells and initiate an inflammatory reaction. This reaction may finally damage the graft. Recombinant P-selectin glycoprotein immunoglobulin is thought to act by blocking these proteins on the cell surface. This is expected to stop the inflammatory process that can damage the graft.

What is the stage of development of this medicine?

The effects of recombinant P-selectin glycoprotein immunoglobulin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with post transplantation graft dysfunction were ongoing.

Recombinant P-selectin glycoprotein immunoglobulin was not authorised anywhere worldwide for post transplantation graft dysfunction 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 5 April 2006 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Recombinant P-selectin glycoprotein immunoglobulin</p>
Active substanceRecombinant P-selectin glycoprotein immunoglobulin
Medicine Name
Disease/conditionPrevention of post transplantation graft dysfunction
Date of decision22/05/2006
OutcomePositive
Orphan decision numberEU/3/06/376

Review of designation

Sponsor’s contact details:

RJM Consultancy Ltd
Alresford House
60 West Street
Farnham, Surrey GU9 7EH
United Kingdom
Telephone: +44 78 76 50 71 00
Telefax: +44 12 52 73 37 80

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.