EU/3/13/1221

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in May 2017 on request of the Sponsor.

On 16 January 2014, orphan designation (EU/3/13/1221) was granted by the European Commission to Chugai Pharma Europe Ltd, United Kingdom, for humanised monoclonal modified IgG4 antibody with bispecific structure targeting factors IX, IXa, X and Xa for the treatment of haemophilia A.

The sponsorship was transferred to Roche Registration Limited, United Kingdom, in December 2014.

This medicine is now known as emicizumab.

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

What is haemophilia A?

Haemophilia A is an inherited bleeding disorder that is caused by the lack of factor VIII, which 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 prolonged bleeding 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 A is a debilitating disease that is life-long and may be life threatening because bleeding can happen in the brain, the spinal cord or the gut.

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

At the time of designation, haemophilia A affected approximately 0.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 36,000 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. At the time of designation, this represented a population of 512,900,000 (Eurostat 2014).

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 might be given less often than existing treatments. In addition, the medicine is expected to be given under the skin (instead of into a vein), which is expected to improve compliance with treatment. These assumptions 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 expected to work on blood clotting by replacing the activity of factor VIII and promoting the activation of another factor called factor X by factor IXa, which starts the clotting process. By activating factor X, this medicine is expected to control the bleeding disorder in patients with haemophilia A because it acts directly on factor X, independently of factor VIII.

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, 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 6 November 2013 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.

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