EU/3/08/554 - orphan designation for treatment of pulmonary arterial hypertension

beraprost sodium

Overview

Please note that this product was withdrawn from the Union Register of orphan medicinal products in January 2020 on request of the sponsor.

On 10 July 2008, orphan designation (EU/3/08/554) was granted by the European Commission to Lung Rx Limited, United Kingdom, for beraprost sodium (modified release tablet) for the treatment of pulmonary arterial hypertension.

The sponsorship was transferred to IDEA Innovative Drugs European Associates Limited, United Kingdom, in February 2012.

In December 2012, Innovative Drug European Associates Limited changed name to IDEA Innovative Drug European Associates Limited.

The sponsorship was transferred to IDEA Innovative Drug European Associates (Ireland) Limited, Republic of Ireland, in February 2019.

What is pulmonary arterial hypertension?

Pulmonary arterial hypertension is a disorder of the blood vessels leading to surrounding the lungs, in which the blood pressure in the pulmonary artery (the large blood vessel that carries blood from the heart to the lungs) becomes high. It is caused by the walls of the lung's blood vessels become thicker, harder and less elastic, making it more difficult for the blood to move through the vessels. The cause of these changes in the vessels is unknown. In the long term, the high blood pressure causes damage to the heart and symptoms such as shortness of breath, tiredness, chest pain, weakness, coughing, wheezing and swelling.

Pulmonary arterial hypertension is a debilitating disease that is long-lasting and may be life-threatening.

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

At the time of designation, pulmonary arterial hypertension affected less than 2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 101,000 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).

---

*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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 502,800,000 (Eurostat 2008).

What treatments are available?

At the time of submission of the application for orphan drug designation, several medicines were authorised in the European Union (EU) for the treatment of pulmonary arterial hypertension.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that beraprost sodium (modified-release tablet) might be of potential significant benefit for the treatment of pulmonary arterial hypertension. This is mainly because of the expected clinical effect of the medicine in relation to and its mode of administration, which could make the treatment more convenient for patients. This assumption will need to be confirmed at the time of marketing authorisation, to maintain the orphan status.

How is this medicine expected to work?

Beraprost sodium is a molecule that is very similar to prostacyclin, a natural substance that causes the blood vessels to dilate (widen). In patients with pulmonary arterial hypertension, beraprost sodium is expected to act in the same way as prostacyclin to dilate the pulmonary arteries, lowering the blood pressure in the pulmonary artery and improving the symptoms of the disease.

Beraprost sodium is to be available as modified-release tablets. This means that the tablets have been made to release beraprost sodium over a few hours. This would allow the patient to be treated using an oral treatment that can be taken twice a day.

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 pulmonary arterial hypertension were ongoing.

Beraprost is authorised in different countries for indications including pulmonary arterial hypertension. A modified-release form of beraprost has been available in Japan for pulmonary arterial hypertension since 2007.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on 14 May 2008 recommending the granting of the abovementioned 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.

EU/3/08/554: Public summary of positive opinion for orphan designation of beraprost sodium (modified release tablet) for the treatment of pulmonary arterial hypertension

Adopted Reference Number: EMA/COMP/369082/2008 Rev.5

Key facts

• Active substance:
  beraprost sodium
• Intended use:
  Treatment of pulmonary arterial hypertension
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/08/554
• Date of designation:
  10/07/2008
• Sponsor:

  IDEA Innovative Drug European Associates (Ireland) Limited; 13 Classon House; Dundrum Business Park, Dundrum; Dublin, D14 W9Y3; Ireland; Tel. + 353 1903 6117; E-mail: [e-mail]

Review of designation

Please note that this product was withdrawn from the Union Register of orphan medicinal products in January 2020 on request of the sponsor.