EU/3/08/561

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Orphan designation

On 5 September 2008, orphan designation (EU/3/08/561) was granted by the European Commission to Kiadis Pharma Netherlands B.V, Netherlands, for donor lymphocyte preparation depleted of functional alloreactive T-cells for the prevention of Graft-versus-Host disease.

What is Graft-versus-Host disease?

Graft-versus-host disease (GvHD) is a complication of bone marrow transplant. The bone marrow is the spongy tissue inside the large bones in the body that makes blood cells and platelets (components that help the blood to clot). Bone marrow transplants are used for diseases such as leukaemia or multiple myeloma (cancer of the white blood cells).

In GvHD, the cells in the transplanted bone marrow react against the patients’ organs, such as the stomach, gut, skin and liver, leading to organ damage. GvHD may happen in the short term, or later after transplantation, in which case a wider range of organs can be involved. GvHD is a serious, life-threatening disease.

What is the estimated number of patients at risk of developing the condition?

At the time of designation the population at risk of developing GvHD was approximately 0.2 in 10,000 people in the European Union (EU)*. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 10,000 people.

*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 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

What methods of prevention are available?

Several products were authorised for the condition in some countries in the Community at the time of submission of the application for orphan drug designation.
Current methods for preventing GvHD aim to reduce the activity of the immune system (the body’s natural defences). These methods work by targeting cells that divide often, such as the immune system cells.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that donor lymphocyte preparation depleted of functional alloreactive T-cells might be of potential significant benefit for the prevention of GvHD, mainly because it has a new mechanism of action. This 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 product is a preparation of a type of immune system cell called T-cells. It involves the use of a chemical compound that reacts to light, and destroys the T-cells in the cell preparation called donor lymphocyte preparation that could attack the patient’s organs.
This cell preparation is given to patients whose immune system is weak after chemotherapy. It is thought to enable the immune system to be rebuilt with healthy T-cells.

What is the stage of development of this medicine?

The effects of donor lymphocyte preparation depleted of functional alloreactive T-cells have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients who have undergone a bone marrow transplant were ongoing.
Donor lymphocyte preparation depleted of functional alloreactive T-cells was not authorised anywhere worldwide for prevention of GvHD at the time of submission. Orphan designation of donor lymphocyte preparation depleted of functional alloreactive T-cells had been granted in the United States of America for immune reconstitution and prevention of GvHD following allogeneic haematopoietic stem cell transplantation.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 11 June 2008 a positive opinion recommending the grant of the above-mentioned 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>Donor lymphocyte preparation depleted of functional alloreactive T-cells</p>
Active substanceDonor lymphocyte preparation depleted of functional alloreactive T-cells
Medicine Name
Disease/conditionPrevention of Graft-versus-Host Disease
Date of decision05/09/2008
OutcomePositive
Orphan decision numberEU/3/08/561

Review of designation

Sponsor’s contact details:

Kiadis Pharma Netherlands B.V
Entrada 231-234
1096 EG Amsterdam
The Netherlands
Telephone: +31 20 3140 250
Telefax: +31 20 3140 251
E-mail: info@kiadis.com

Patients’ association contact point:

Not available