EU/3/00/001

Wasting is defined as the unintended loss of bodyweight and it is one of the symptoms of AIDS (acquired immune deficiency syndrome). There are usually several causes for this symptom such as loss of appetite, diarrhoea, decreased absorption of nutrients from food and immune factors (a decreased activity of the body's natural defence system which is also the main symptom of AIDS). Irrespective of the cause, wasting leads to widespread metabolic and immunologic dysfunction. AIDS wasting is chronically debilitating.

At the time of designation AIDS wasting affected less than 2 in 10,000 people in the European Union (EU)*. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 76,000 people.

At the time of submission of application for orphan drug designation, there were no medicinal products authorised for the treatment of AIDS wasting. Some patients were treated with appetite-stimulating agents.

Somatropin consists of growth hormone, which is a natural substance found in the body. It is thought that one of the causes of wasting in AIDS patients is a decrease in growth hormone production or a decrease in sensitivity towards this hormone in the body. Growth hormone has many functions in various tissues but it primarily stimulates the growth of muscle tissue (lean body mass) and the break down of fatty tissues.

The effects of somatropin were evaluated in experimental models.

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

Somatropin was authorised in the United States and Japan for the treatment of AIDS wasting, at the time of submission. Orphan designation of the medicinal product was granted in the United States for this condition.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 11 July 2000 a positive opinion recommending the grant of the above-mentioned designation.

• 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 European Union) 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.