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Orphan designation

On 14 September 2007, orphan designation (EU/3/07/481) was granted by the European Commission to Astion Pharma A/S, Denmark, for R-salbutamol sulphate for the treatment of cutaneous forms of lupus erythematosus.

What are cutaneous forms of lupus erythematosus?

Lupus erythematosus is a disease where the defence system of the body (immune system) damages its own tissues, such as the skin and those forming the joints, and organs, such as the kidneys. Inflamed lesions of the skin caused by lupus erythematosus can be divided into three subgroups: discoid lupus erythematosus (DLE), subacute cutaneous lupus erythematosus (SCLE), and other local lupus erythematosus (lupus erythematosus profundus and lupus panniculitis). Lesions occur predominantly in areas exposed to sunlight, such as the face, scalp, neck and upper extremities. A small number of patients develop scattered lesions, affecting widespread areas including the trunk and limbs. Generalized involvement is milder in SCLE, and in the other forms there is involvement of the deeper tissues beneath the skin. The condition may persist for years, with repeated recurrences, and may be chronically debilitating.

What are the methods of treatment available?

Several products with anti-inflammatory activity and some antimalarial drugs were authorised for the condition in some countries in the Community at the time of submission of the application for orphan drug designation. R-salbutamol sulphate could be of potential significant benefit for the treatment of cutaneous forms of lupus erythematosus, in particular with regards to improved efficacy. This assumption of significant benefit will have to be confirmed at the time of marketing authorisation, as this is necessary to maintain the orphan status.

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

Based on the information provided by the sponsor and previous knowledge of the Committee, cutaneous forms of lupus erythematosus was considered to affect not more than 5 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 249,000 persons in total.

*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 Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

R-salbutamol sulphate selectively binds to and activates β2-adrenoceptors, which are molecules on the surface of many cells. This results in a decreased function of genes that induce inflammation. In short, the product has a general anti-inflammatory effect, and is administered directly on the skin as a cream.

What is the stage of development of this medicinal product?

The effects of R-salbutamol sulphate were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with cutaneous forms of lupus erythematosus were ongoing.

R-salbutamol sulphate was not authorised anywhere in the world for the treatment of cutaneous forms of lupus erythematosus, nor designated as an orphan medicinal product for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 25 July 2007 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition, 
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for 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 will be necessary before this product can be granted a marketing authorisation.

Key facts

Product details for <p>R-salbutamol sulphate</p>
Active substanceR-salbutamol sulphate
Medicine Name
Disease/conditionTreatment of cutaneus forms of lupus erythromatosus
Date of decision14/09/2007
Orphan decision numberEU/3/07/481

Review of designation

Sponsor’s contact details:

Astion Pharma A/S
Fruebjergvej 3
2100 Copenhagen Ø
Telephone: +45 4333 4800
Telefax: +45 4333 4801

Patients’ associations contact points:

LF : Lupus France
7 Rue de Rocroy
75010 Paris
Telephone: +33 1 45 26 33 27
Telefax: +33 1 45 26 33 27

Lupus UK
St James House
Eastern Road, Romford
Essex RM1 3NH
United Kingdom
Telephone: +44 1708 73 12 51
Telefax: +44 1708 73 12 52

Lupus Erythematodes Selbsthilfegemeinschaft e.V.
Döppersberg 20
42103 Wuppertal
Telephone: +49 202 49 68 797
Telefax: +49 202 49 68 798