This medicine is now known as remdesivir.
On 17 February 2016, orphan designation (EU/3/16/1615) was granted by the European Commission to Gilead Sciences International Ltd, United Kingdom, for 2-ethylbutyl (2S)-2-{[(S)-{[(2R,3S,4R,5R)-5-(4-aminopyrrolo[2,1-f][1,2,4]triazin-7-yl)-5-cyano-3,4-dihydroxytetrahydrofuran-2-yl]methoxy}(phenoxy)phosphoryl]amino}propanoate for the treatment of Ebola virus disease.
The sponsorship was transferred to Gilead Sciences Ireland UC in June 2018.
Ebola virus disease is a severe disease caused by infection with viruses known as ebolaviruses. There are 5 known species of ebolavirus, 4 of which are known to cause the disease in humans. Zaire ebolavirus, sometimes referred to simply as 'ebola virus' or EBOV, is the cause of the largest outbreaks of the disease to date and has led to the most deaths.
Infection is caused by contact with the body fluids of an infected person. After infection there is an incubation period of between 2 to 21 days, following which the newly infected person starts to experience symptoms. The first symptoms typically are fever, headache, fatigue, muscle pain and sore throat. These are followed by other symptoms such as diarrhoea, vomiting, rash, kidney and liver problems and, in some cases, internal bleeding and bleeding from the gums, eyes, nose and ears. Patients are infectious once they start to develop symptoms.
Ebola virus disease is a life-threatening condition that is frequently fatal due to fluid loss and severe bleeding.
At the time of designation, Ebola virus disease affected less than 0.01 in 10,000 people in the European Union (EU). This 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 application for orphan designation, there were no satisfactory treatments authorised in the EU for treating Ebola virus disease. Different treatments were used to relieve the symptoms of the disease, such as providing fluids by a drip into a vein and balancing electrolytes (body salts), maintaining oxygen supply to the body and blood pressure, and treating other infections if they develop.
Ebolaviruses contain RNA (a type of genetic material). In order for ebolaviruses to multiply and cause disease, copies of the RNA must be made by a virus enzyme called RNA polymerase. This medicine is expected to block the RNA polymerase of the Ebola virus and so to prevent ebolaviruses from multiplying.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, a clinical trial with the medicine in healthy volunteers was ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for Ebola virus disease. Orphan designation of the medicine had been granted in the United States for Ebola virus disease.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 January 2016 recommending the granting of this designation.
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.
Gilead Sciences Ireland UC
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: