EU/3/01/024 - orphan designation for treatment of haemorrhagic fever with renal syndrome
Ribavirin
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in February 2009 on request of the sponsor.
On 14 February 2001, orphan designation (EU/3/01/024) was granted by the European Commission to ICN Pharmaceuticals Limited, United Kingdom, for ribavirin for the treatment of haemorrhagic fever with renal syndrome. ICN Pharmaceuticals Limited subsequently changed name to Valeant Pharmaceuticals Ltd.
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.
Key facts
- Active substance
- Ribavirin
- Intended use
- Treatment of haemorrhagic fever with renal syndrome
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/01/024
- Date of designation
- Sponsor
Valeant Pharmaceuticals Ltd
Cedarwood, Chineham Business Park
Crockford Lane
Basingstoke RG24 8WD
United Kingdom
Telephone: +44 1256 70 77 44
Telefax: +44 1256 70 73 34
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: