EU/3/16/1685

On 27 June 2016, orphan designation (EU/3/16/1685) was granted by the European Commission to Serendex Pharmaceuticals A/S, Denmark, for molgramostim 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, an organ close to the stomach) or trauma 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 3.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 170,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 513,700,000 (Eurostat 2016).

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 treated with surfactant preparations but they were not authorised for the condition. A surfactant lines the airways and allows gases to pass easily between the lung and blood. Healthy lungs produce their own surfactant.

How is this medicine expected to work?

This medicine is made of molgramostim to be given by inhalation. Molgramostim is a copy of a human protein known as granulocyte macrophage colony-stimulating factor (GM-CSF), which stimulates the immune system. How it would work in acute respiratory distress syndrome is not clear but it appears to act by repairing the cells lining the lung and clearing out microbes. In clinical studies in patients with severe lung conditions, molgramostim treatment improved the flow of oxygen into the blood.

What is the stage of development of this medicine?

The effects of molgramostim have been evaluated in experimental models.

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

At the time of submission, molgramostim given by injection was authorised in the EU for increasing neutrophils (a type of white blood cell) in certain conditions.

At the time of submission, molgramostim was not authorised anywhere in the EU for acute respiratory distress syndrome 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 19 May 2016 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.