EU/3/01/029 - orphan designation for treatment of adenovirus infection in immunocompromised patients
ribavirin
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in September 2007 on request of the sponsor.
On 8 March 2001, orphan designation (EU/3/01/029) was granted by the European Commission to ICN Pharmaceuticals Limited, United Kingdom, for ribavirin for the treatment of adenovirus infection in immunocompromised patients.
The sponsor changed name to Valeant Pharmaceuticals Limited, United Kingdom, in June 2004.
Patients whose immune system has been impaired by disease or treatment are immunocompromised. Their immune system's ability to fight infections is compromised or entirely absent and these patients are easily infected by micro-organisms such as adenoviruses.
Adenovirus disease in immunocompromised patients has been attributed to both primary infection and reactivation of endogenous latent infection (virus is sleeping in the body after the first infection). In immunocompromised patients adenovirus infections can cause lesions (injuries) in the lung, the pancreas, the gastrointestinal tract, the liver, the kidney and the urinary system. Furthermore, it can cause throat infections and spread via the blood stream. Infection of adenovirus in immunocompromised patients can be chronically debilitating and life threatening.
At the time of designation, adenovirus infection in immunocompromised patients affected approximately 0.6 to 3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 23,000 to 113,000 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).
No medicinal products were authorised for the treatment of adenovirus infection in immunocompromised patients in the Community at the time of submission of the application for orphan drug designation.
Ribavirin is a purine nucleoside analogue. The purine nucleosides are part of the fundamental genetic material of human cells but also of viruses. Ribavirin stops the adenovirus from copying its genetic material (blocks adenovirus' replication) and thus prevents the virus from multiplying itself and spreading.
The effects of ribavirin were evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in immunocompromised patients with adenovirus infection were completed.
Ribavirin was not marketed anywhere worldwide for treatment of adenovirus infection in immunocompromised patients or designated as orphan medicinal product elsewhere for this condition, at the time of submission.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 15 January 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.
Key facts
- Active substance
- ribavirin
- Intended use
- Treatment of adenovirus infection in immunocompromised patients
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/01/029
- Date of designation
- Sponsor
Valeant Pharmaceuticals Ltd
Cedarwood, Chineham Business Park
Crockford Lane
Basingstoke RG24 8WD
United Kingdom
Telephone: +44 1256 70 77 44
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: