EU/3/07/505 - orphan designation for treatment of acute respiratory distress syndrome (ARDS)

Interferon beta

Overview

Please note that this product was withdrawn from the Union Register of orphan medicinal products in February 2023 on request of the Sponsor.

On 27 June 2018, 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 respiratory distress syndrome.

What is acute respiratory distress syndrome?

Acute respiratory distress syndrome is a condition in which lung injury leads to inflammation and fluid in the air sacs in the lungs, resulting in insufficient oxygen passing into the blood. The lungs eventually become stiff with scar tissue and breathing becomes very difficult.

There are many possible causes of acute respiratory distress syndrome, including: inhaling high concentrations of smoke, harmful substances, or oxygen; severe burns; blood infection; pneumonia (infection of the lungs); pancreatitis (inflammation of the pancreas); or damage to other parts of the body. Symptoms occur suddenly and include fast shallow breathing, shortness of breath, and severe tiredness.

Acute respiratory distress syndrome is a life-threatening condition because of the worsening problems with breathing.

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

At the time of designation, acute respiratory distress syndrome affected approximately 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 181,000 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).

 

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*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 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).

What treatments are available?

At the time of designation, there were no authorised products for the treatment of acute respiratory distress syndrome in the EU. Patients affected by the condition were put on a ventilator (a machine that 'breathes' for the patient) and were given treatment for the underlying cause for the condition.

How is this medicine expected to work?

Inflammation associated with acute respiratory distress syndrome makes blood vessels in the lung to become leaky. Interferon beta has an anti-inflammatory action; it is expected to reduce the leakage of blood and fluids from the blood vessels, thereby reducing the damage to the lungs in patients with the condition.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with acute respiratory distress syndrome were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for acute respiratory distress syndrome or designated as an orphan medicinal product elsewhere for this condition.

This medicine had been designated orphan on 29 November 2007 for the treatment of acute lung injury. At the request of the sponsor and having assessed the additional information submitted, the COMP adopted an opinion on 24 May 2018 recommending the change of the orphan condition from acute lung injury to acute respiratory distress syndrome, in accordance to the most recent disease classification.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:

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/3/07/505: Public summary of positive opinion of acute respiratory distress syndrome

Adopted Reference Number: EMA/396437/2018

Key facts

• Active substance:
  Interferon beta
• Intended use:
  Treatment of acute respiratory distress syndrome (ARDS)
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/07/505
• Date of designation:
  29/11/2007
• Sponsor:

  Faron Pharmaceuticals Limited; Joukahaisenkatu 6; 20520 Turku; Finland; Tel. [phone]; Fax [phone]; E-mail: [e-mail]