EU/3/03/179 - orphan designation for treatment of hereditary factor-XIII deficiency

Recombinant human factor XIII (composed of two A subunits)

Overview

Please note that this product (marketed as NovoThirteen) was withdrawn from the Community Register of designated orphan medicinal products in July 2012 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.

On 12 December 2003, orphan designation (EU/3/03/179) was granted by the European Commission to Chiltern International Limited, United Kingdom, for recombinant human factor XIII (composed of two A subunits) for the treatment hereditary factor-XIII deficiency.

The sponsorship was transferred to Novo Nordisk A/S, Denmark, in December 2004.

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

What is hereditary factor-XIII deficiency?

Factor-XIII (FXIII) deficiency is an inherited blood disorder characterised by abnormal blood clotting that may result in abnormal bleeding. FXIII is an enzyme that helps to stabilise the blood clot by mechanically linking certain big molecules to one another and thereby increasing the mechanical strength of blood clots. In affected individuals, the blood clot is not strong enough, resulting in longer bleeding time and poor wound healing. Blood may seep into surrounding tissues, resulting in local pain and swelling. Internal bleeding may occur. Factor XIII deficiency can be life-threatening, since in a significant proportion of affected individuals bleeding in the brain tissue (intracranial haemorrhage) occurs.

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

At the time of designation, hereditary factor XIII deficiency affected approximately 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of number of around 380 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. At the time of designation, this represented a population of 382,800,000 (Eurostat 2003).

What treatments are available?

Several products to treat factor XIII deficiency have been authorised in a few Member States of the European Union. These products contain different levels of FXIII. Recombinant human FXIII might be of potential significant benefit for the treatment of hereditary FXIII deficiency, due in particular to the increased availability in all Member States. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Recombinant human FXIII is expected to replace the missing enzyme and thus it should prevent the longer bleeding times and the bleeding into the surrounding tissue in patients with hereditary FXIII deficiency.

What is the stage of development of this medicine?

The effects of recombinant human FXIII have been evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in patients with hereditary FXIII deficiency were ongoing.

Recombinant human FXIII was not marketed anywhere worldwide for treatment of hereditary FXIII deficiency, at the time of submission.

Orphan designation of recombinant human factor XIII was granted in United States for the condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 November 2003 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;
• either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for 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 will be necessary before this product can be granted a marketing authorisation.

EU/3/03/179: Public summary of positive opinion for orphan designation of recombinant human factor XIII (composed of two A subunits) for the treatment of hereditary factor-XIII deficiency

AdoptedReference Number: EMA/COMP/1590/2003, Rev. 3

Key facts

• Active substance:
  Recombinant human factor XIII (composed of two A subunits)
• Medicine name:
  NovoThirteen
• Intended use:
  Treatment of hereditary factor-XIII deficiency
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/03/179
• Date of designation:
  12/12/2003
• Sponsor:

  Novo Nordisk A/S; Novo Alle 1; 2880 Bagsvaerd; Denmark; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

Please note that this product (marketed as NovoThirteen) was withdrawn from the Community Register of designated orphan medicinal products in July 2012 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.