On 29 November 2007, orphan designation (EU/3/07/505) was granted by the European Commission to Faron Pharmaceuticals Limited, Finland, for Interferon beta for the treatment of acute lung injury.
- What is acute lung injury?
Acute lung injury (ALI) is a condition in which the capacity of the lungs to oxygenate the blood is greatly reduced, even if oxygen is administered in high concentrations. The most severe form of ALI is known as ARDS (adult respiratory distress syndrome).
The lungs are made up of tiny air sacs, called alveoli, which is the location of oxygen and carbon dioxide exchange between air and blood. When an infection or another disease injures the lungs, blood and fluid begin to leak from the small blood vessels (capillaries) that surround the alveoli, into the alveoli. When this happens, air cannot enter the alveoli, which means that the normal functions of the lung tissue are impaired. This leads to inflammation (a response to the injury caused to the tissue) and progressive formation of scar tissue in the walls of the alveoli. The patient will experience an increasing shortness of breath. There are many possible causes of ALI such as inhaling high concentrations of smoke, toxins, or oxygen; severe burns; blood infections (the most common cause); lung infections; or trauma to other parts of the body. ALI is a life-threatening condition.
- What are the methods of treatment available?
No medicinal products were authorised in the Community for the treatment of ALI or ARDS at the time of submission of the application for orphan drug designation. The treatment options for ALI were limited to life-support and artificial ventilation with high concentrations of oxygen. Antibiotics were also used to treat the infections and reduce the inflammation.
- 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, ALI was considered to affect 3.4 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 169,000 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?
Interferon beta has a known anti-inflammatory action; in addition, it is expected to reduce the leakage of blood and fluids from the capillaries in the lungs (and elsewhere in the body). Interferon beta is thus expected to reduce the damage to the lungs, and improve the oxygenation of the patient.
- What is the stage of development of this medicinal product?
The effects of interferon beta were evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials in patients with ALI had been initiated.
Interferon beta was not authorised anywhere in the world for the treatment of ALI, or designated as orphan medicinal product elsewhere for this condition, at the time of submission.
Intereferon beta is authorised in the European Union for the treatment of multiple sclerosis.According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 10 October 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.
|Name||Language||First published||Last updated|
|EU/3/07/505: Public summary of positive opinion of interferon beta for the treatment of acute lung injury||(English only)||02/07/2008|
|Active substance||Interferon beta|
|Disease/condition||Treatment of acute lung injury|
|Date of decision||28/11/2007|
|Orphan decision number||EU/3/07/505|
Review of designation
Sponsor’s contact details:
Faron Pharmaceuticals Limited
Tykistökatu 6 B
Telephone: +358 2 78 8401
Telefax: +358 2 478 8411
Patients’ association contact point: