EU/3/15/1501 - orphan designation for treatment of haemophilia B

Adeno-associated viral vector containing the human factor IX gene

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in September 2017 on request of the sponsor.

On 19 June 2015, orphan designation (EU/3/15/1501) was granted by the European Commission to Baxalta Innovations GmbH, Austria, for adeno-associated viral vector containing the human factor IX gene for the treatment of haemophilia B.

What is haemophilia B?

Haemophilia B is an inherited bleeding disorder that is caused by the lack of factor IX, which 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. This can lead to permanent injury if it happens repeatedly.

Haemophilia B is a debilitating disease that is life long 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 0.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 10,000 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).

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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 512,900,000 (Eurostat 2015).

What treatments are available?

At the time of designation, medicines containing factor IX were authorised in the EU for the treatment of haemophilia B, to replace the missing 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 factor VIIa (the activated form of factor VII, another protein involved in blood clotting), either alone or as part of a combination treatment.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with haemophilia B. Data from early studies in experimental models showed that the medicine reduces bleeding without causing the body to produce the inhibitors that limit the use of some current 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?

The medicine is made of a virus that has been modified to contain the gene for factor IX, which is lacking in patients with haemophilia B. After being given once to the patient as an injection into a vein, the virus is expected to carry the factor-IX gene into the liver cells, enabling them to produce the missing factor IX for an extended period of time. This is expected to control the bleeding disorder.

The type of virus used in this medicine ('adeno-associated virus') does not cause disease in humans.

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, a clinical trial with the medicine in patients with haemophilia B was ongoing.

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

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 May 2015 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/3/15/1501: Public summary of opinion on orphan designation: Adeno-associated viral vector containing the human factor IX gene for treatment of haemophilia B

AdoptedReference Number: EMA/COMP/360279/2015

Key facts

• Active substance:
  Adeno-associated viral vector containing the human factor IX gene
• Medicine name:
  -
• Intended use:
  Treatment of haemophilia B
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/15/1501
• Date of designation:
  19/06/2015
• Sponsor:

  Baxalta Innovations GmbH; Industriestrasse 67; A-1221 Vienna; Austria; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in September 2017 on request of the sponsor.