EU/3/19/2176 - orphan designation for treatment of haemophilia A

Recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein

Overview

On 28 June 2019, orphan designation EU/3/19/2176 was granted by the European Commission to Swedish Orphan Biovitrum AB (publ), Sweden, for recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein (also known as BIVV001) for the treatment of haemophilia A.

What is haemophilia A?

Haemophilia A is an inherited bleeding disorder caused by the lack of factor VIII, which is one of the proteins involved in blood coagulation (clotting). Patients with haemophilia A are prone to bleeding and bleed for a long time after injury or surgery. Bleeding can also happen within muscles or 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 occur in the brain, spinal cord or gut.
 

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

At the time of designation, haemophilia A affected approximately 0.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 41,000 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).

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*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 28), Norway, Iceland and Liechtenstein. This represents a population of 518,400,000 (Eurostat 2019).

What treatments are available?

At the time of designation, medicines containing factor VIII were authorised in the EU for the treatment of haemophilia A, to replace the missing protein. However, factor VIII medicines did not work in some patients with haemophilia A because the immune system (the body’s natural defences) can produce ‘inhibitors’ (antibodies) against factor VIII which stop the factor VIII medicine from working. In these cases, different blood clotting factors or other treatments needed to be used, either alone or as part of a combination treatment.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with haemophilia A because preliminary results indicate that it can increase how long factor VIII remains in the blood. 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?

Patients with haemophilia A cannot make enough functioning factor VIII because the gene for producing the clotting factor is damaged.

This medicine is made up of a genetically engineered factor VIII protein attached to fragments from other proteins: von Willebrand factor and XTEN. When injected into the patient, it is expected that the proteins attached to factor VIII will make factor VIII remain longer in the bloodstream, thereby reducing the tendency for bleeding for a long time.
 

What is the stage of development of this medicine?

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

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

At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of haemophilia A. Orphan designation had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a Positive opinion on 23 May 2019, 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/3/19/2176: Public summary of opinion on orphan designation: Recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein for the treatment of haemophilia A

AdoptedReference Number: EMADOC-628903358-1041Summary:

On 28 June 2019, orphan designation EU/3/19/2176 was granted by the European Commission to Swedish Orphan Biovitrum AB (publ), Sweden, for recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein (also known as BIVV001) for the treatment of haemophilia A.

Key facts

• Active substance:
  Recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein
• Intended use:
  Treatment of haemophilia A
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/19/2176
• Date of designation:
  28/06/2019
• Sponsor:

  Swedish Orphan Biovitrum AB (publ); Stockholm; 112 76 Stockholm; Sweden; Tel. [phone]; E-mail: [e-mail]