EU/3/05/317

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Orphan designation

On 26 August 2005, orphan designation (EU/3/05/317) was granted by the European Commission to Henogen SA, Belgium, for a mixture of anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein and anti-CD7 mAb (WT1)-ricin A chain fusion protein for the treatment of graft-versus-host-disease.

The sponsorship was transferred to Xenikos BV, The Netherlands, in January 2010.

What is graft-versus-host-disease?

The bone marrow is the spongy tissue inside the large bones in the body. The bone marrow makes red blood cells (which carry oxygen and other materials to all tissues of the body), white blood cells (which fight infection), and platelets (which make the blood clot). Bone marrow transplantation (replacing with healthy marrow) is a treatment used against certain diseases of the bone marrow. A frequent complication of bone-marrow transplantation is the development of a disease called graft-versus-host-disease (GvHD). This disease involves a reaction between the donor cells and the recipient's native tissues leading to injury of the recipient’s tissues. GvHD occurs in acute and chronic form. The organs most commonly affected in acute GvHD are the stomach and the intestines, the skin, and the liver. Chronic GvHD involves a much wider range of tissues than the acute form. The condition is chronically debilitating and life-threatening.

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

At the time of designation, graft-versus-host-disease affected approximately 0.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 19,000 people*, and is below the threshold 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 466,600,000 (Eurostat 2005).

What treatments are available?

The methods of treatment authorised for GvHD in the Community, at the time of submission of the application for orphan designation, consisted of certain steroid hormones (corticosteroids, a group of chemical substances, which modulate the activity of certain organs and of the immune system) administered at high doses. Other therapies include drugs that inhibit the immune response (immunosuppressants). A mixture of anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein and anti-CD7 mAb (WT1)-ricin A chain fusion protein might be of potential significant benefit for the treatment of GvHD, particularly in terms of a selective action against those cells that are responsible for the disease. This assumption remains to be proven. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

A mixture of anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein and anti-CD7 mAb (WT1)-ricin A chain fusion protein is a combination of a toxic substance with an antibody. Antibodies are proteins that specifically recognise and attach themselves to cell structures such as proteins found on the surface of the cells. In the case of this antibody, the target is a protein (CD3 and CD7) found on certain cells of the immune system, so-called mature T cells. The antibody is expected to bind to these cells and to induce their destruction, by release of the toxin into the cells. This might thereby prevent GvHD.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, clinical trials in patients with GvHD were ongoing.

A mixture of anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein and anti-CD7 mAb (WT1)-ricin A chain fusion protein was not marketed anywhere worldwide for treatment of GvHD or designated as an 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 13 July 2005 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 European Union) 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>A mixture of anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein and anti-CD7 mAb (WT1)-ricin A chain fusion protein</p>
Active substanceA mixture of anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein and anti-CD7 mAb (WT1)-ricin A chain fusion protein
Medicine Name
Disease/conditionTreatment of graft-versus-host disease
Date of decision26/08/2005
OutcomePositive
Orphan decision numberEU/3/05/317

Review of designation

Sponsor’s contact details:

Xenikos BV
Molenveldlaan 152 A
6523RN Nijmegen
The Netherlands
Tel. +31 24 300 01 00
Fax +31 84 741 07 35

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.