EU/3/08/565

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Orphan designation

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.

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 is 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).

*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. This represents a population of 504,800,000 (Eurostat 2009).

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.

Key facts

Product details for <p>Drotrecogin alfa (activated)</p>
Active substanceDrotrecogin alfa (activated)
Medicine Name
Disease/conditionTreatment of acute respiratory distress syndrome
Date of decision22/09/2008
OutcomePositive
Orphan decision numberEU/3/08/565

Review of designation

Sponsor’s contact details:

Drugrecure Aps
c/o Serendex ApS
Cobis Building
Ole Maaløes vej 3
2200 Copenhagen
Denmark
Telephone: + 45 43 58 37 59
Telefax: + 45 43 58 37 69
E-mail: fiala@serendex.com

Patients’ association(s) contact point(s):

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.