EU/3/13/1104 - orphan designation for treatment of systemic sclerosis

Terguride

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in October 2013 on request of the sponsor.

On 8 February 2013, orphan designation (EU/3/13/1104) was granted by the European Commission to High Tech Participations GmbH, Germany, for terguride for the treatment of systemic sclerosis.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

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).

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*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 509,000,000 (Eurostat 2013).

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 high blood pressure in the lungs 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?

Terguride mainly blocks 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 fibrosis. In the blood-vessel walls, 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 on the cells in the blood vessel walls, preventing serotonin from attaching to them. This is expected to block the normal action of serotonin, thereby reducing the abnormal growth of 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, no 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 9 January 2013 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.

EU/3/13/1104: Public summary of positive opinion of terguride for the treatment of systemic sclerosis

AdoptedReference Number: EMA/COMP/27796/2013 Rev.1

Key facts

• Active substance:
  Terguride
• Intended use:
  Treatment of systemic sclerosis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/13/1104
• Date of designation:
  08/02/2013
• Sponsor:

  High Tech Participations GmbH; Kreuzstrasse 34; D 40210 Düsseldorf; Germany; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in October 2013 on request of the sponsor, before a marketing authorisation had been granted.