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Orphan designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in September 2014 on request of the sponsor.

On 3 June 2008, orphan designation (EU/3/08/552) was granted by the European Commission to Novo Nordisk A/S, Denmark, for pegylated recombinant factor VIIa for the treatment of haemophilia B.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is haemophilia B?

Haemophilia B is an inherited disorder where the body’s ability to control blood clotting (coagulation) is impaired. It is caused by an inborn deficiency (low levels) of a substance called factor IX, which is one of the human proteins involved in the blood clotting process. Patients with haemophilia B have longer bleeding times than normal and poor wound healing after injury or surgery. Bleeding can also happen within muscles or the spaces in the joints, such as the elbows, knees and ankles, which can lead to permanent injury if it happens repeatedly. Rare, but life-threatening bleeding can also happen in the brain and spinal cord, the throat or the gut.

Haemophilia B is a debilitating disease that is long lasting and may be life threatening.

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

At the time of designation, haemophilia B affected approximately 0.1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 5,000 people*, and 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).

* 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.
At the time of designation, this represented a population of 502,800,000 (Eurostat 2008).

What treatments are available?

Several products containing factor IX are authorised for the treatment of haemophilia B in the European Union. These are used to replace the missing protein. However, not all patients with haemophilia B can benefit from these products because the immune system can react against them by producing inhibitors (antibodies) against factor IX.

In these cases, other treatments need to be used, such as medicines containing other coagulation factors, either alone or in combination. These can include factor VIIa, which works by activating another factor called factor X to start the coagulation process. Factor VIIa acts directly on factor X, independently from factor IX, so medicines containing factor VIIa can be of use in patients who have developed inhibitors to factor IX.

The sponsor has submitted satisfactory documentation to justify its assumption that pegylated recombinant factor VIIa might be of benefit for the treatment of haemophilia B, because it might contribute to the care of the haemophilia B patients who have developed inhibitors against factor IX and it may possibly be given less often than the currently used treatment. This assumption will need to be confirmed at the time of marketing authorisation to maintain the orphan status.

How is this medicine expected to work?

Pegylated recombinant factor VIIa is expected to work in the body in the same way as human factor VIIa. By activating factor X, it is expected to control the bleeding disorder in patients who have developed inhibitors to factor IX.

Pegylated recombinant factor VIIa 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 factor VIIa. It has also been modified by a process called ‘pegylation’, meaning that it has been coated with a chemical called polyethylene glycol. This decreases the rate at which the substance is removed from the body and allows the medicine to be given less often than other products that are available.

What is the stage of development of this medicine?

The effects of pegylated recombinant factor VIIa were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials were ongoing.

At the time of submission, pegylated recombinant factor VIIa was not authorised anywhere in the world for the treatment of haemophilia B or designated as 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 8 April 2008 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 or not of alternative methods of diagnosis, prevention or treatment and
  • either the rarity of the condition (considered to affect not more than five in 10,000 people in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are products that are still under investigation, which are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Pegylated recombinant factor VIIa</p>
Active substancePegylated recombinant factor VIIa
Medicine Name
Disease/conditionTreatment of haemophilia B
Date of decision03/06/2008
Orphan decision numberEU/3/08/552

Review of designation

Sponsor’s contact details:

Novo Nordisk A/S
Novo Allé
Tel. +45 44 44 88 88
Fax +45 44 49 05 55

Patients' organisations

  • 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.