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Orphan designation

On 24 January 2013, orphan designation (EU/3/12/1096) was granted by the European Commission to Serodapharm GmbH, Germany, for terguride for the treatment of systemic sclerosis.

What is systemic sclerosis?

Systemic sclerosis is a complex disease in which the immune system (the body’s natural defences) is overactivated, causing inflammation and excess production of various proteins, particularly collagen. The reason why the immune system is overactivated is not known. Collagen is an important component of connective tissue (the tissue that supports the skin and internal organs).

The overproduction of collagen leads to the abnormal growth of connective tissue, causing the skin to become thick and hard. It can also damage tissues in the blood-vessel walls of the internal organs, such as the heart, lungs and kidneys. This makes it more difficult for the blood to move through the vessels, causing tissue damage, circulation problems and high blood pressure.

Systemic sclerosis is a long-lasting debilitating disease and may be life-threatening because of its possible effects on the gut, heart, lungs and kidneys.

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

At the time of designation, systemic sclerosis affected approximately 1.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 81,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

What treatments are available?

At the time of designation, there were no treatments for systemic sclerosis that could stop the build-up of collagen. Treatments authorised in the EU were aimed at relieving the symptoms of the disease and limiting the damage it causes. Several medicines were used to reduce inflammation and circulation problems. Bosentan was authorised in the EU specifically to treat patients with systemic sclerosis who have pulmonary arterial hypertension (high blood pressure in the artery that leads from the heart to the lung) or digital ulcers (sores on the fingers and toes).

The sponsor has provided sufficient information to show that terguride might be of significant benefit for patients with systemic sclerosis because it works in a different way to existing treatments by targeting the disease itself rather than its consequences, and early studies show that it may reduce the build-up of connective tissue. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

In systemic sclerosis, terguride is expected to work mainly by blocking the action of serotonin, a neurotransmitter (a substance that allows nerve cells to communicate with each other) which is found in high amounts in patients with systemic sclerosis. Serotonin attaches to certain receptors on the surfaces of cells and is involved in the production of fibrous connective tissue. Terguride is expected to attach to the receptors for serotonin, preventing serotonin from attaching to them. This is expected to block the action of serotonin, thereby reducing the abnormal growth of fibrous connective tissue.

What is the stage of development of this medicine?

The effects of terguride have been evaluated in experimental models.

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

At the time of submission, terguride was not authorised anywhere in the EU for systemic sclerosis or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 December 2012 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 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.

Key facts

Product details for <p>Terguride</p>
Active substanceTerguride
Medicine Name
Disease/conditionTreatment of systemic sclerosis
Date of decision24/01/2013
Orphan decision numberEU/3/12/1096

Review of designation

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

Sponsor’s contact details:

Serodapharm GmbH
Königin-Luise-Str. 27
D-14195 Berlin
Tel. +49 302 8869 878
Fax +49 302 8869 696

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.