EU/3/11/863 - orphan designation for treatment of haemophilia B

recombinant fusion protein linking human coagulation factor VIIa with human albumin

Overview

On 13 May 2011, orphan designation (EU/3/11/863) was granted by the European Commission to CSL Behring GmbH, Germany, for recombinant fusion protein linking human coagulation factor VIIa with human albumin for the treatment of haemophilia B.

What is haemophilia B?

Haemophilia B is an inherited bleeding disorder that is caused by the lack of a substance called factor IX. Factor IX is one of the proteins involved in the blood coagulation (clotting) process. Patients with haemophilia B are more prone to bleeding than normal and have 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.

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

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 is 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 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 treatments are available?

At the time of submission of the application for orphan designation, medicines containing factor IX were authorised in the EU for the treatment of haemophilia B. These medicines were used to replace the missing factor IX protein. However, not all patients with haemophilia B could benefit from these medicines because the immune system (the body's natural defences) can react against them by producing 'inhibitors' (antibodies) against factor IX. In these cases, other treatments needed to be used, such as other coagulation factors such as factor VIIa, either alone or as part of a combination treatment.

The sponsor has provided sufficient information to show that recombinant fusion protein linking human coagulation factor VIIa with human albumin might be of significant benefit for patients with haemophilia B because it could be used in haemophilia B patients who have developed inhibitors against factor IX and it might be given less often than existing treatments. 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?

Recombinant fusion protein linking human coagulation factor VIIa with human albumin is made of a copy of human factor VIIa, which is attached to albumin, a protein that acts as a carrier. The medicine is expected to work on blood clotting in the same way as human factor VIIa. In the body, factor VIIa is involved in blood clotting. It activates another factor called factor X, which starts the clotting process. By activating factor X, this medicine is expected to control the bleeding disorder in patients who have developed inhibitors to factor IX because it acts directly on factor X, independently of factor IX.

Attaching albumin to factor VIIa is expected to decrease the rate at which the factor VIIa is cleared from the body, allowing more time between injections than for medicines that contain only factor VIIa.

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 factor VIIa linked to albumin.

What is the stage of development of this medicine?

The effects of recombinant fusion protein linking human coagulation factor VIIa with human albumin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with haemophilia B had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for haemophilia B 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 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.

EU/3/11/863: Public summary of opinion on orphan designation: Recombinant fusion protein linking human coagulation factor VIIa with human albumin for the treatment of haemophilia B

Adopted Reference Number: EMA/COMP/158306/2011

Key facts

• Active substance:
  recombinant fusion protein linking human coagulation factor VIIa with human albumin
• Intended use:
  Treatment of haemophilia B
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/11/863
• Date of designation:
  13/05/2011
• Sponsor:

  CSL Behring GmbH; Emil-von-Behring-Str. 76; 35041 Marburg; Germany; Tel. [phone]; E-mail: [e-mail]

Review of designation

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