EU/3/01/053 - orphan designation for treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Beraprost sodium
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in February 2004 on request of the sponsor.
On 18 September 2001, orphan designation (EU/3/01/053) was granted by the European Commission to Aventis Pharma SA, France, for beraprost sodium for the treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension.
Please note that beraprost sodium is now owned by Toray Industries, Japan.
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. An increase of the number of smooth muscle cells in the walls of small lung arteries (a phenomenon called proliferation) that are remodelling the vessels, may lead to obstructions in the microcirculation, which will then lead 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 which has been brought to its site by the blood current), which directly leads to pulmonary hypertension. Pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension are chronically debilitating and life-threatening.
One medicinal product was authorised for the treatment of pulmonary arterial hypertension in the Community at the time of submission of the application for orphan drug designation. Beraprost sodium might be of potential significant benefit for the treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension mainly because it might contribute to patient care because of the new route of administration. This potential benefit will have to be
confirmed at the time of marketing authorisation and this will be necessary to maintain the orphan status.
According to the information provided by the sponsor, pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension was considered to affect about 26,000 in the European Union.
*Disclaimer: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001) and may differ from the true number of patients affected by the condition. This estimate is based on available information and calculations presented by the sponsor at the time of the application.
Beraprost sodium is a protein similar to a natural substance, prostacyclin, normally released by certain white blood cells (cells of the body's immune system). It is formulated in an oral tablet and might enlarge the diameter of the vessels, also on the pulmonary arteries. This effect is called vasodilatation.
Beraprost sodium might also help to diminish the blood clotting in the vessels, which might also help in the treatment of the condition.
The effects of beraprost sodium were evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension were ongoing.
Beraprost sodium was not marketed anywhere worldwide for the treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension, at the time of submission.
Orphan designation of beraprost sodium was granted in Japan and in the United states for the same condition.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 18 July 2001 a positive opinion recommending the grant of the above-mentioned designation.
- 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
- Active substance
- Beraprost sodium
- Intended use
- Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/01/053
- Date of designation
- Sponsor
Aventis Pharma SA
20 avenue Raymond Aron
F- 92165 Antony Cedex
France
Telephone: +33 1 55 71 62 57
Telefax: +33 1 55 71 78 54
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: