EU/3/03/169 - orphan designation for treatment of dermatomyositis

Human immunoglobulin

Overview

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in September 2013 on request of the sponsor.

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

On 20 October 2003, orphan designation (EU/3/03/169) was granted by the European Commission to Orfagen, France, for human immunoglobulin for the treatment of dermatomyositis.

What is dermatomyositis?

Dermatomyositis is a disease involving the muscles and the skin. The typical sign of dermatomyositis is progressive weakness of the muscles of the upper part of the legs or arms. In the course of the disease also the muscles of the oesophagus, the respiratory system and the heart can be affected so that the patients have difficulties in eating and breathing. The disease is also associated with characteristic severe symptoms in the skin. Patients affected by dermatomyositis have a higher risk of developing cancer and should be accurately examined on a regular basis.

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

At the time of designation, dermatomyositis affected less than 1.25 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 48,000 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. At the time of designation, this represented a population of 382,800,000 (Eurostat 2003).

What treatments are available?

Several products including glucocorticoids and azathioprine were authorised for the condition in some countries in the Community at the time of submission of the application for orphan-drug designation. Human immunoglobulin is expected to be of potential significant benefit in particular in patients not responding to or not tolerating the standard treatments. The 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?

Although the exact cause of the disease remains unknown, it seems that the immune system of the patient is directly involved. The patient's immune system seems, in fact, to react against some natural body elements. The human-immunoglobulin mechanism of action is complex. It seems to interfere with the immune system and decrease the immune reactions against body elements.

What is the stage of development of this medicine?

Human immunoglobulin has been used in the treatment of dermatomyositis for several years. Case reports and results from small clinical trials on the use of human immunoglobulin in dermatomyositis have been published in the literature.

At the time of submission of the application for orphan designation, no clinical trials in patients with the condition were ongoing.

The proposed medicinal product is not authorised in any country in the designated indication, but it is authorised in the Community for the treatment of immunoglobulin deficiency and some auto-immune diseases. It was designated for treatment of dermatomyositis and polymyositis in the United States in October 1993.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 September 2003 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/3/03/169: Public summary of positive on orphan designation of human immunoglobulin for the treatment of dermatomyositis

AdoptedReference Number: EMA/COMP/1442/2004 Rev. 2

Key facts

• Active substance:
  Human immunoglobulin
• Intended use:
  Treatment of dermatomyositis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/03/169
• Date of designation:
  20/10/2003
• Sponsor:

  Orfagen; CRDPF Langlade; 3 Avenue Hubert Curien - BP 13562; 31035 Toulouse Cedex 1; France; Tel. [phone]; Fax [phone]; E-mail [e-mail]

Review of designation

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