EU/3/12/1047

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Orphan designation

On 10 October 2012, orphan designation (EU/3/12/1047) was granted by the European Commission to Tarix Pharmaceuticals Limited, Cyprus, for Asp-Arg-Val-Tyr-Ile-His-Pro for the treatment of acute lung injury.

The sponsorship was transferred to Gregory Fryer Associates Ltd, United Kingdom, in June 2013.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

What is acute lung injury?

Acute lung injury is damage to the lungs that causes fluid to leak into the alveoli (the tiny air sacs in the lungs where the exchange of oxygen between the air and the blood takes place). This results in the blood not receiving enough oxygen. Patients with acute lung injury have difficulty breathing, tachycardia (rapid heartbeat), and their lips, fingers and toes become slightly blue in colour. There are many possible causes of acute lung injury, such as inhaling high amounts of smoke or toxic gases, severe burns, near-drowning, drug overdose, blood or lung infections, inflammation of the pancreas, lung contusion (bruising) and trauma to another part of the body.

Acute lung injury is a life-threatening disease because it causes breathing problems, which can be fatal, and is associated with multiple organ failure and sepsis (when bacteria and their toxins circulate in the blood and damage the organs).

What is the estimated number of patients?

At the time of designation, acute lung injury affected not more than 3.4 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 173,000 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 509,000,000 (Eurostat 2012).

What treatments are available?

At the time of designation, the main treatment for acute lung injury was mechanical ventilation (using a mask or a tube inserted into the airways to help the patient to breathe). Patients were also given treatment for the causes of lung injury including antibiotics to treat infections, and medicines to control fluids in the body and symptoms such as pain. In the United Kingdom, prednisolone (a steroid anti-inflammatory medicine) is authorised for lung problems, and may be given in some cases of acute lung injury, but this is not a usual treatment.

The sponsor has provided sufficient information to show that Asp-Arg-Val-Tyr-Ile-His-Pro might be of significant benefit for patients with acute lung injury because it works in a different way to existing treatments and early studies show that it might be more effective at treating the condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Asp-Arg-Val-Tyr-Ile-His-Pro is a peptide (a short chain of amino acids, the building blocks of proteins), which is a copy of a naturally occurring peptide called angiotensin (1-7) found in the blood. In patients with acute lung injury, Asp-Arg-Val-Tyr-Ile-His-Pro is expected to restore the balance of the ‘renin-angiotensin system’, a hormonal system that regulates blood pressure and fluid balance, which is believed to be over-activated in the lungs of these patients. This medicine is thought to work by attaching to receptors in the small blood vessels in the lungs, which has a number of effects that reduce the local actions of the renin-angiotensin system.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of Asp-Arg-Val-Tyr-Ile-His-Pro in experimental models was ongoing.

At the time of submission, no clinical trials with this medicine in patients with acute lung injury had been started.

At the time of submission, Asp-Arg-Val-Tyr-Ile-His-Pro was not authorised anywhere in the EU for acute lung injury or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 September 2012 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;
  • 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.

Key facts

Product details for <p>Asp-Arg-Val-Tyr-Ile-His-Pro</p>
Active substanceAsp-Arg-Val-Tyr-Ile-His-Pro
Medicine Name
Disease/conditionTreatment of acute lung injury
Date of decision10/10/2012
OutcomePositive
Orphan decision numberEU/3/12/1047

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details:

Gregory Fryer Associates Ltd
30 St Thomas Place
Cambridgeshire Business Park
Ely
Cambridgeshire CB7 4EX
United Kingdom
Tel. +44 (0)13 5364 5590
Fax +44 (0)13 5364 5599
E-mail: info@gf-associates.co.uk

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases see:

  • Orphanet, a database containing information on rare diseases which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.