EU/3/20/2301: Orphan designation for the treatment of acute respiratory distress syndrome (ARDS)

Acute respiratory distress syndrome (ARDS) 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; transfusion of high amounts of blood products; 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.

• 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.

At the time of designation, ARDS affected approximately 3.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 166,000 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).

*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

At the time of designation, corticosteroid medicines were authorised for treating ARDS in some EU countries. Physical methods and other medicines were used to help patients with their breathing and to reduce some symptoms.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with ARDS because laboratory studies showed that it works in a different way to reduce build-up of fluid in the lungs and improve the amount of oxygen passing into the blood.

The medicine contains a human hormone called adrenomedullin, which has several effects in the body including reducing inflammation and stopping blood vessels becoming leaky. By reducing inflammation and leakage of fluid from the blood vessels into the lung tissue, the medicine is expected to help the lungs work normally and allow oxygen to enter the blood more efficiently in patients with ARDS.

This medicine is made up of adrenomedullin attached to a chain of polyethylene glycol (PEG) to make it remain in the body for longer. It is expected to be used through a nebuliser, a device that creates a fine spray of the medicine for the patient to breathe in.

The effects of pegylated adrenomedullin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with pegylated adrenomedullin in patients with ARDS had been started.

At the time of submission, pegylated adrenomedullin was not authorised anywhere in the EU for the treatment of ARDS or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 18 June 2020, recommending the granting of this designation.