EU/3/08/565 - orphan designation for treatment of acute respiratory distress syndrome (ARDS)

drotrecogin alfa (activated)

Overview

On 22 September 2008, orphan designation (EU/3/08/565) was granted by the European Commission to Drugrecure Aps, Denmark, for drotrecogin alfa (activated) for the treatment of acute respiratory distress syndrome.

The sponsorship was transferred to Savara ApS, Denmark  in September 2016. The sponsor’s address was updated in November 2019.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in September 2021 on request of the Sponsor.

What is acute respiratory distress syndrome?

In Acute Respiratory Distress Syndrome (ARDS) the lungs are injured and this results in respiratory failure. There are many possible causes of the type of lung injury that leads to ARDS. These include inhaling high concentrations of smoke, toxins, or oxygen; severe burns; blood infection; pneumonia (infection of the lungs); pancreatitis (inflammation of the pancreas, an organ close to the stomach) or trauma to other parts of the body.

Lungs contain alveoli, which are tiny air sacs where the oxygen is passed into the blood. In ARDS blood and fluid begin to leak into the alveoli. When this happens, oxygen cannot enter the alveoli, which means oxygen is no longer getting into the blood. Because the lungs are inflamed and filled with fluid, the patient finds it increasingly difficult to breathe. The inflammation (swelling and redness) in the lungs leads to scarring and vascular damage including formation of small clots. The lungs eventually become stiff with scar tissue and breathing becomes very difficult. ARDS is life-threatening because it makes breathing extremely difficult and can cause death.

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

At the time of designation ARDS affected less than 3.5 in 10,000 people in the European Union (EU). This was below the threshold for orphan designation, which is 5 people in 10,000, and is equivalent to a total of around 176,000 people*. This is based on the information provided by the sponsor and 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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 502,800,000 (Eurostat 2008).

What treatments are available?

There were no authorised products for the condition in the Community at the time of submission of the application for orphan drug designation.

Existing treatments for patients affected by the condition included surfactant preparations that were used by not authorised for ARDS.

How is this medicine expected to work?

Drotrecogin alfa (activated) is a recombinant human Activated Protein C (rhAPC) with similar properties as the Activated Protein C produced by the body. Activated protein C (APC) prevents blood from clotting. Blood clotting can worsen the situation in ARDS when this happens in small vessels which become obstructed and further reduce oxygen exchange. This medicinal product is expected to act as the natural APC, stop blood clotting and preventing this way the worsening of the process that brings about ADRS.

What is the stage of development of this medicine?

The effects of activated drotrecogin alfa were evaluated in experimental models.

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

Activated drotrecogin alfa was not authorised anywhere worldwide for acute respiratory distress syndrome 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 9 July 2008 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 European Union) 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/3/08/565: Public summary of positive opinion for orphan designation of drotrecogin alfa (activated) for the treatment of acute respiratory distress syndrome

Adopted Reference Number: EMEA/COMP/441380/2008 Rev. 3

Key facts

• Active substance:
  drotrecogin alfa (activated)
• Intended use:
  Treatment of acute respiratory distress syndrome (ARDS)
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/08/565
• Date of designation:
  22/09/2008
• Sponsor:

  Savara ApS; Amaliegade 10; Copenhagen K; 1256; Tel: [phone]; E-mail: [e-mail]