EU/3/01/034: Orphan designation for the treatment of Wegener's granulomatosis

Wegener's granulomatosis is a disease characterised by chronic inflammation of the blood vessels affecting the respiratory tract (sinuses, nose, trachea (windpipe) and lungs) and kidneys. This inflammation can damage the organs (partial or complete obstruction of the lung) by limiting the blood flow to them and destroying normal tissue. Wegener's granulomatosis is considered as a life threatening and chronically debilitating condition.

At the time of designation, approximately 0.4 to 0.6 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 15,000 to 23,000 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).

At the time of submission of the application for the orphan drug designation there were some medicinal products authorised for the condition in some member states of the Community.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that gusperimus trihydrochloride might be of potential significant benefit for the treatment of Wegener's granulomatosis. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

The exact way gusperimus trihydrochloride works in the body is not fully understood. Gusperimus trihydrochloride is a chemically synthesised immunosuppressive agent (a compound that reduces the activity of the immune system). The medicinal product interferes with an intracellular signalling event, thus inhibiting growth and differentiation of certain cell of the immune system and, in this way, it is expected to reduce the symptoms of Wegener's granulomatosis.

The effects of gusperimus trihydrochloride were evaluated in experimental models.

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

At the time of submission, gusperimus trihydrochloride was not marketed anywhere worldwide for the treatment of Wegener's granulomatosis or designated as 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 February 2001 recommending the granting of this designation.

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.