EU/3/10/783

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Orphan designation

On 20 September 2010, orphan designation (EU/3/10/783) was granted by the European Commission to Biogen Idec Limited, United Kingdom, for recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1 for the treatment of haemophilia A.

What is haemophilia A?

Haemophilia A is an inherited bleeding disorder that is caused by the lack of a substance called factor VIII. Factor VIII is one of the proteins involved in the blood coagulation (clotting) process. Patients with haemophilia A are more prone to bleeding than normal and have poor wound healing after injury or surgery. Bleeding can also happen within muscles or within the spaces in the joints, such as the elbows, knees and ankles. This can lead to permanent injury if it happens repeatedly.

Haemophilia A is a debilitating disease that is life-long and may be life threatening because bleeding can happen in the brain and the spinal cord, the throat or the gut.

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

At the time of designation, haemophilia A affected approximately 0.6 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 30,000 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 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 506,500,000 (Eurostat 2010).

What treatments are available?

At the time of submission of the application for orphan drug designation, medicines containing factor VIII were authorised in the EU for the treatment of haemophilia A, to replace the missing protein. Some patients needed other treatments, such as medicines containing other substances involved in blood clotting.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with haemophilia A because it is expected to be given less often than the treatments that are currently being used. 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 made of a copy of human factor VIII, which is attached to a part of a human antibody called IgG1. In the body, the medicine is expected to replace the missing factor VIII, making the patient less prone to bleeding. The IgG1 part allows the medicine to attach to receptors on the surface of immune system cells. This allows the factor VIII to remain in the blood for longer before being cleared from the body. This is expected to allow more time between injections than for medicines that contain factor VIII on its own.

The medicine is made by a method known as ‘recombinant DNA technology’: it is made by a cell that has received a gene (DNA) that makes it able to produce this fusion molecule.

What is the stage of development of this medicine?

The effects of this medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with this medicine in patients with haemophilia A were ongoing.

At the time of submission, this medicine was not authorised anywhere in the EU for haemophilia A or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 2 June 2010 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>Recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1</p>
Active substanceRecombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1
Medicine Name
Disease/conditionTreatment of haemophilia A
Date of decision20/09/2010
OutcomePositive
Orphan decision numberEU/3/10/783

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:

Biogen Idec Limited
Innovation House
70 Norden Road
Maidenhead
Berkshire SL6 4AY
United Kingdom
Telephone: + 44 1628 501 000
Telefax: + 44 1628 501 010
ukreception@biogenidec.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.