EU/3/03/173 - orphan designation for treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension

vasoactive Intestinal Peptide

Overview

On 22 December 2003, orphan designation (EU/3/03/173) was granted by the European Commission to Mondobiotech Laboratories Anstalt, Liechtenstein, for vasoactive intestinal peptide for the treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension.

The sponsorship was transferred to Biogen Idec Limited, United Kingdom, in December 2006 and subsequently to Mondobiotech Laboratories AG, Fürstentum Liechtenstein, in June 2010.

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. An increase of smooth muscle cells in the lung microcirculation (so called cell proliferation) are remodelling the vessels and may lead to obstructions in the microcirculation, which will then lead to an increase in the pressure.

Chronic thromboembolic pulmonary hypertension is a complication representing less than 1% of all cases of acute pulmonary embolism (the sudden blocking of an 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.

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

At the time of designation, pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension affected not more than 2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 78,000 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: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 385,000,000 (Eurostat 2002) and may differ from the true number of patients affected by the condition.

What treatments are available?

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.

Vasoactive intestinal peptide might be of potential significant benefit for the treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension in particular with regards to the new mechanism of action. The preliminary preclinical and clinical results indicate that this product may also offer a major contribution to patient care because of the new route of administration and a different safety profile. This benefit will have to be confirmed at the time of marketing authorisation and will be necessary to maintain the orphan status.

How is this medicine expected to work?

It has vasodilating (enlargement of the vessel) properties, including an effect on pulmonary arteries. Furthermore it inhibits the proliferation of muscle cells in the vessels and in the bronchi. Vasoactive intestinal peptide has also anti-platelet properties, which may play a beneficial role in preventing embolism (platelets are important for blood coagulation).

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 and chronic thromboembolic pulmonary hypertension were ongoing.

Vasoactive intestinal peptide was not marketed anywhere worldwide for pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 October 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 of alternative methods of diagnosis, prevention or treatment and
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the Community) 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/3/03/173: Public summary of positive opinion for orphan designation of vasoactive intestinal peptide for the treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension

Adopted Reference Number: EMEA/COMP/1537/03 Rev. 3

Key facts

• Active substance:
  vasoactive Intestinal Peptide
• Intended use:
  Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/03/173
• Date of designation:
  22/12/2003
• Sponsor:

  Mondobiotech Laboratories AG; Herrengasse 21; 9490 Vaduz; Fürstentum Liechtenstein; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]