EU/3/01/024: Orphan designation for the treatment of haemorrhagic fever with renal syndrome

Haemorrhagic fever with renal syndrome is a disease characterised by fever, kidney failure and bleeding. In severe cases patients may suffer from shock. Humans can contract the disease by inhalation of particles from secretions (urine, droppings and saliva) of rodents (such as mice and rats) infected with viruses, called Hantavirus. The disease is not known to transmit from one person to another. The disease is potentially life threatening and chronically debilitating condition.

At the time of designation, haemorrhagic fever with renal syndrome affected approximately 3.7 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 139,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

No medicinal products were authorised for the treatment of haemorrhagic fever with renal syndrome in the Community at the time of submission of the application for orphan drug designation.

Ribavirin is structurally similar to purine nucleosides. The purine nucleosides are part of the fundamental genetic material of human cells but also of viruses. Ribavirin inhibits the replication (the copying of the genetic material) of viruses causing haemorrhagic fever with renal syndrome. Therefore it is thought to prevent the viruses from multiplying and causing harm, ultimately limit the disease.

The effects of ribavirin were evaluated in experimental models. At the time of submission of the application for orphan designation, no clinical trials in patients with haemorrhagic fever with renal syndrome were initiated.

Ribavirin received orphan drug designation in the United States in 1991 for the treatment of haemorrhagic fever with renal syndrome.

Ribavirin was not marketed or designated as orphan medicinal product in the EU for the treatment of haemorrhagic fever and renal syndrome, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 December 2000 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.