EU/3/15/1558

On 9 October 2015, orphan designation (EU/3/15/1558) was granted by the European Commission to Dr Stefan Blesse, Germany, for three chimeric human/murine monoclonal antibodies against the Ebola (Zaire) surface glycoprotein (also known as ZMapp) for the treatment for Ebola virus disease.

What is Ebola virus disease?

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 develop symptoms.

Ebola virus disease is a life-threatening condition that is frequently fatal due to fluid loss and severe bleeding.

What is the estimated number of patients affected by the condition?

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 isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

What treatments are available?

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.

How is this medicine expected to work?

This medicine is made of 3 monoclonal antibodies (type of proteins) that have been designed to recognise and attach to different parts of a protein on the ebolavirus surface. By attaching to the virus, the antibodies are expected to neutralise its action.

What is the stage of development of this medicine?

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 patients with Ebola virus disease 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 this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 September 2015 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• 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.

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.