EU/3/18/2002 - orphan designation for treatment of Crimean-Congo haemorrhagic fever
Ribavirin
OrphanHuman
Overview
On 21 March 2018, orphan designation (EU/3/18/2002) was granted by the European Commission to Pharmadev Healthcare Ltd, Ireland, for ribavirin for the treatment of Crimean-Congo haemorrhagic fever.
The sponsor’s address was updated in April 2021.
Crimean-Congo haemorrhagic fever is a viral infection that causes several symptoms, including high temperatures, muscle pain, vomiting and diarrhoea, and bleeding from the skin and internal membranes.
The disease is caused by a virus called nairovirus, which is passed on to humans by tick bites. People can also be infected by contact with blood or body parts of animals that have the virus.
Crimean-Congo haemorrhagic fever is debilitating and life-threatening, with around 13% of people who become infected dying from it.
At the time of designation, Crimean-Congo haemorrhagic fever affected less than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 500 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).
*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).
At the time of the orphan designation there were no medicines or vaccines authorised for Crimean-Congo haemorrhagic fever in the EU. Treatment aimed at relieving the symptoms of the disease and included fluids and blood products.
Ribavirin is an antiviral medicine that is already authorised for treating another viral infection, chronic hepatitis C. The medicine is known as a 'nucleoside analogue' and is thought to work by interfering with the virus genetic material, which viruses need to survive and multiply.
The effects of ribavirin have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with ribavirin in patients with Crimean-Congo haemorrhagic fever were ongoing.
At the time of submission, ribavirin was not authorised anywhere in the EU for Crimean-Congo haemorrhagic fever or designated as an 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 15 February 2018 recommending the granting of this 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 EU) 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.
Key facts
- Active substance
- Ribavirin
- Intended use
- Treatment of Crimean-Congo haemorrhagic fever
- Orphan designation status
- Positive
- EU designation number
- EU/3/18/2002
- Date of designation
- Sponsor
Pharmadev Healthcare Ltd
The Black Church
Saint Mary's Place North
Dublin 7
Dublin D07 P4AX
Ireland
Tel. +353 56 77 06 537
E-mail: jlbarnoux@pharmadevhealthcare.eu
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
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: