EU/3/11/891

  • Email
  • Help

Orphan designation

On 5 August 2011, orphan designation (EU/3/11/891) was granted by the European Commission to Emergent Sales and Marketing Germany GmbH, Germany, for human anthrax monoclonal antibody for post-exposure prophylaxis of inhalation anthrax disease.

What is inhalation anthrax disease?

Anthrax is a severe disease caused by infection with bacteria called Bacillus anthracis. The bacteria produce spores that are very resistant and can lay ‘dormant’ (inactive) until they find an organism where they can develop and multiply. Anthrax commonly affects animals such as sheep and cows, but can spread to humans when they are exposed to spores from infected animals or contaminated animal products.

The most severe type of anthrax is inhalation anthrax, which occurs when a person has breathed in the bacteria’s spores. The first symptoms of inhalation anthrax are similar to a cold. Several days after the spores have been inhaled, they grow into new bacteria and start to release toxins, which cause internal bleeding, swelling and the death of tissue.

Inhalation anthrax disease is a life threatening disease because, if not treated early, it leads to the accumulation of fluid in the lungs, severe inflammation and bleeding of the tissues in the chest and meningitis (inflammation of the membranes that surround the brain and spine).

What is the estimated number of patients at risk of developing the condition?

At the time of designation, the number of patients at risk of inhalation anthrax disease was estimated to be approximately 0.01 people in 10,000 in the European Union (EU)*. This is equivalent to a total of around 500 people, which 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

What methods of prevention are available?

At the time of designation, several antibiotics were authorised in the EU for the prevention of inhalation anthrax disease following exposure to B. anthracis.

The sponsor has provided sufficient information to show that human anthrax monoclonal antibody might be of significant benefit for patients at risk of inhalation anthrax disease, because it works in a different and more targeted way to existing treatments and early studies in experimental models indicate that it might improve the prevention of inhalation anthrax disease in people who have been exposed to B. anthracis. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

This medicine is a monoclonal antibody, a type of protein that has been designed to recognise and attach to the anthrax toxins. The medicine is expected to be injected into patients who are at risk of developing anthrax after having been exposed to B. anthracis. By attaching to the anthrax toxins, the medicine is expected to stop them from entering into the body’s cells, thereby preventing the disease from developing.

What is the stage of development of this medicine?

The effects of human anthrax monoclonal antibody 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 post-exposure prophylaxis for inhalation anthrax disease. Orphan designation of human anthrax monoclonal antibody had been granted in the United States of America for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 February 2011 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.

Key facts

Product details for <p>Human anthrax monoclonal antibody</p>
Active substanceHuman anthrax monoclonal antibody
Medicine Name
Disease/conditionFor post-exposure prophylaxis of inhalation anthrax disease
Date of decision05/08/2011
OutcomePositive
Orphan decision numberEU/3/11/891

Review of designation

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

Sponsor’s contact details:

Emergent Sales and Marketing Germany GmbH
Walter-Gropius-Str. 17
80807 München
Germany
Telephone: +49 89 550 69 88 66
Telefax: +49 89 550 69 88 99
E-mail: lockharts@ebsi.com

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.