EU/3/18/2027 - orphan designation for treatment of Ebola virus disease
three human monoclonal antibodies against the EBOV glycoprotein
OrphanHuman
Overview
On 25 May 2018, orphan designation (EU/3/18/2027) was granted by the European Commission to Regeneron Ireland U.C., Ireland, for three human monoclonal antibodies against the EBOV glycoprotein (also known as REGN3470-3471-3479) for the treatment of Ebola virus disease.
In December 2019, Regeneron Ireland U.C. changed name to Regeneron Ireland DAC.
The sponsor’s address was updated in October 2021.
Ebola virus disease is a severe disease caused by infection with 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 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 through severe diarrhoea and severe bleeding.
At the time of designation, Ebola virus disease affected less than 0.001 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 50 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).
*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 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.
This medicine is made of 3 monoclonal antibodies, proteins that have been designed to recognise and attach to different parts of a target substance on the surface of the virus. By attaching to the virus, the antibodies are expected to prevent it from infecting cells and reproducing itself, and to stimulate the immune system (the body's natural defences) to kill the virus and infected cells.
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 had been completed.
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 the treatment of Ebola virus infection.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 April 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
- three human monoclonal antibodies against the EBOV glycoprotein
- Intended use
- Treatment of Ebola virus disease
- Orphan designation status
- Positive
- EU designation number
- EU/3/18/2027
- Date of designation
- Sponsor
Regeneron Ireland DAC
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: