EU/3/07/443

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Orphan designation

On 20 March 2007, orphan designation (EU/3/07/443) was granted by the European Commission to Proteo Biotech AG, Germany, for elafin for the treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension.

What is pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension?

Pulmonary arterial hypertension is a rare blood vessel disorder of the lung in which the pressure in the pulmonary artery (the blood vessel that leads from the heart to the lungs) rises above normal levels. The number of smooth muscle cells in the walls of small lung arteries increases (a phenomenon called proliferation) resulting in remodelling of these vessels. This may lead to obstructions in the microcirculation and then to an increase in the blood pressure.

Chronic thromboembolic pulmonary hypertension is a complication representing less than 1% of all cases of acute pulmonary embolism (the sudden blocking of a lung artery by a clot or foreign material that has been transported by the blood), directly leading to pulmonary hypertension. Pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension are chronically debilitating and life-threatening.

What are the methods of treatment?

Several medicinal products were authorised for the treatment of pulmonary arterial hypertension in the Community at the time of submission of the application for orphan drug designation.

Elafin might be of potential significant benefit for the treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension in particular with regards to improved efficacy based on the new mechanism of action. This benefit will have to be confirmed at the time of marketing authorisation and will be necessary to maintain the orphan status.

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

Based on the information provided by the sponsor and previous knowledge of the Committee, pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension was considered to affect 2.2 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 110,000 persons.

* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

One of the mechanisms that contribute to pulmonary arterial hypertension is the body’s ability to increase the thickness of the pulmonary vessels. In pulmonary arterial hypertension this ability is not well regulated and the vessels become narrower. The disease worsens as the inner space of the vessels become smaller and it becomes increasingly difficult for the blood to move though the vessels.

Elafin is a protein that has an inhibitory activity over endogenous vascular elastase. Endogenous vascular elastase is a protein that has an important activity in the process of thickening of the blood vessel wall. By inhibiting this protein, elafin is expected to stop the thickening of the blood vessel wall, seen in pulmonary arterial hypertension.

What is the stage of development of this medicinal product?

At the time of submission, the effects of elafin were evaluated in experimental models.

Elafin was not authorised anywhere worldwide for pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension, 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 7 February 2007 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition,
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • 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.

Key facts

Product details for <p>Elafin</p>
Active substanceElafin
Medicine Name
Disease/conditionTreatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of decision19/03/2007
OutcomePositive
Orphan decision numberEU/3/07/443

Review of designation

Sponsor’s contact details:

Proteo Biotech AG
Am Kiel Kanal 44
D-24106 Kiel
Germany
Telephone: + 49 431 8888 462
Telefax: + 49 431 8888 463
E-mail: info@proteo.de

Patients’ associations contact points:

Pulmonary Hypertension Association
PO Box 2760
Lewes
Sussex
BN8 4WA
United Kingdom
Telephone: +44 800 389 81 56
Telefax: +44 70 10 71 57 23
E-mail: pha_uk@hotmail.com

HTAP France : Association Française d'aide aux patients souffrant d'Hypertension Artérielle
Pulmonaire
31 Rue Jacques Cellier
51100 Reims
France
Telephone: +33 3 26 36 93 32
E-mail: secretariat@htapfrance.com

PH : Pulmonale Hypertonie e.V.
Wormser Str. 20
76287 Rheinstetten
Germany
Telephone: +49 49 72 42 72 94
Telefax: +49 72 42 95 26 67
E-mail: pphev@aol.com