EU/3/09/690

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Orphan designation

On 9 November 2009, orphan designation (EU/3/09/690) was granted by the European Commission to Emergent Sales and Marketing Germany GmbH, Germany, for human anthrax immunoglobulin for the post-exposure prophylaxis (prevention) of inhalation anthrax disease.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 2 September 2009 recommending the granting of this designation.

What is inhalation anthrax disease?

Anthrax is a severe disease caused by infection with bacteria called Bacillus anthracis (B. 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 of the lungs 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 0.02 people in 10,000 in the European Union (EU)*. This is equivalent to a total of around 1,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP).

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 immunoglobulin might be of significant benefit for patients at risk of inhalation anthrax disease, because 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?

Human anthrax immunoglobulin is a solution of polyclonal antibodies, proteins naturally found in the blood that help the body to fight infections and other diseases. These antibodies have been extracted from the blood of people who have been vaccinated against B. anthracis bacteria. When injected into a patient who is at risk of developing anthrax after inhaling B. anthracis bacteria spores, the antibodies in the medicine are expected to attach to and help to destroy the bacteria, preventing the symptoms of the disease.

What is the stage of development of this medicine?

The effects of human anthrax immunoglobulin have been evaluated in experimental models.
At the time of submission of the application for orphan designation, a clinical trial in healthy volunteers was ongoing.
At the time of submission, human anthrax immunoglobulin was not authorised anywhere in the EU for post-exposure prophylaxis of inhalation anthrax disease or designated as orphan medicinal product elsewhere for this condition.

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 Community) 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 immunoglobulin</p>
Active substanceHuman anthrax immunoglobulin
Medicine Name
Disease/conditionPost-exposure prophylaxis of inhalation anthrax disease
Date of decision08/11/2009
OutcomePositive
Orphan decision numberEU/3/09/690

Review of designation

Sponsor’s contact details:

Emergent Sales and Marketing Germany GmbH
Walter-Gropius Strasse 17
80807 München
Germany
Telephone: + 49 89 550 6988 66
Telefax: + 89 550 6988 99
E-mail: hartmanna@ebsi.com