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Orphan designation

On 3 November 2006, orphan designation (EU/3/06/413) was granted by the European Commission to Dr Falk Pharma GmbH, Germany, for budesonide (oral use) for the treatment of Graft-versus-Host disease.

What is Graft-versus-Host disease?

The bone marrow is the spongy tissue inside the large bones in the body. Bone marrow produces red blood cells (which carry oxygen and other materials to all tissues of the body), white blood cells (that fight infection), and platelets (that make the blood clot). Bone marrow transplantation (replacing with healthy marrow) is a treatment used against certain diseases of the bone marrow. Development of Graft-versus-Host disease (GvHD) is a frequent complication of bone marrow transplantation. 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 less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 46,000 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 based on data from the European Union (EU 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

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), administered at high doses. Other therapies included drugs that inhibit the immune response (immunosuppressants).

How is this medicine expected to work?

Budesonide belongs to the group of corticosteroids, chemicals that affect the activity of certain organs and of the immune system. This medicinal product contains budesonide, which is intended for use as an oral rinse and it is expected to be absorbed through the oral mucosa. This way, the product is expected to act locally, while its side effects are expected to be limited.

What is the stage of development of this medicine?

The effects of budesonide (oral use) were evaluated in experimental models.

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

Budesonide (oral use) was not authorised anywhere worldwide for Graft versus Host Disease, 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 4 October 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>Budesonide (oral use)</p>
Active substanceBudesonide (oral use)
Medicine Name
Disease/conditionTreatment of Graft-versus-Host Disease
Date of decision02/11/2006
Orphan decision numberEU/3/06/413

Review of designation

Sponsor’s contact details:

Dr Falk Pharma GmbH
Leinenweberstrasse 5
D-79041 Freiburg i. Br
Telephone: +49 76 11 51 40
Telefax: +49 76 11 51 43 21

Patients’ association(s) contact point(s):

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.