EU/3/05/333

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Orphan designation

On 14 December 2005, orphan designation (EU/3/05/333) was granted by the European Commission to DKA Consult ApS, Denmark, for eptacog alpha (activated) for the treatment of diffuse alveolar haemorrhage.

The sponsorship was transferred to Pharmaorigin ApS, Denmark, in May 2007.

What is diffuse alveolar haemorrhage?

Tiny air sacs called alveoli are located at the tips of the lungs. The alveoli are responsible for exchanging oxygen and carbon dioxide between air and blood, thus for breathing. Diffuse alveolar haemorrhage is a condition characterized by diffuse bleeding of the alveoli. The manifestations of alveolar hemorrhage include expectoration of blood, difficult respiration and lack of oxygenation of blood. The episodes of bleeding can be sometimes intermittent and limited. The cause of the bleeding is in most of the cases related to an underlying disease. These diseases can be autoimmune diseases, which are conditions in which an individual’s defense system (immune system) starts reacting against his or her own tissues, or infections. In other cases, diffuse alveolar haemorrhage occurs following a bone marrow transplantation (a treatment consisting in replacing abnormal cells of the bone marrow with healthy cells) or as a consequence of the use of some drugs.

The bleeding in the lungs brings disturbances in the capacity of the lungs to work properly, which necessitate controlled intubations and mechanical ventilation. If the bleeding is not stopped, this will lead to permanent reduced functioning of the lungs. Diffuse alveolar haemorrhage is a life threatening condition.

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

At the time of designation, diffuse alveolar haemorrhage affected less than 0.1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 5,000 people, and is below the threshold 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 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are available?

No satisfactory methods exist that were authorised at the time of the application. As initial supportive treatment, the management is focused to stop the bleeding as soon as possible and to restore the lung functioning by intubations and mechanical ventilation. Further management depends mainly on the nature of the underlying disease.

How is this medicine expected to work?

Coagulation factors are a group of proteins present in blood and needed to clot the blood. Eptacog alpha is the activated form of the coagulation factor called factor VII. It is expected that eptacog alpha (activated), delivered directly in the lungs via inhalation, might help to stop the bleeding in the lungs by initiation of the clotting mechanism.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, clinical trials in patients with diffuse alveolar haemorrhage were ongoing.

Eptacog alpha (activated) was not authorised anywhere worldwide for diffuse alveolar haemorrhage or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 24 October 2005 a positive opinion recommending the grant of the above-mentioned 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 European Union) 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.

Key facts

Product details for <p>Eptacog alfa (activated)</p>
Active substanceEptacog alfa (activated)
Medicine Name
Disease/conditionTreatment of diffuse alveolar haemorrhage
Date of decision13/12/2005
OutcomePositive
Orphan decision numberEU/3/05/333

Review of designation

Sponsor’s contact details:

Pharmaorigin ApS
c/o Serendex ApS
Cobis Building
Ole Maaløes vej 3
2200 Copenhagen
Denmark
Telephone: +45 4358 3759
Telefax: +45 4358 3769
E-mail: Fiala@Serendex.com

Patients’ association contact point:

Not available