EU/3/00/016: Orphan designation for the treatment of acute respiratory distress syndrome (ARDS)

Alveoli are tiny air sacs in the lungs where the oxygen is passed into the blood. When the lungs are injured by infection or disease, 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 in the lungs leads to scarring. The lungs eventually become stiff with scar tissue and breathing becomes very difficult. There are many possible causes of the type of lung injury that leads to acute respiratory distress syndrome. These include inhaling high concentrations of smoke, toxins, or oxygen; severe burns; blood infection; pneumonia (inflammation in the lungs); pancreatitis (inflammation of the pancreas, an organ close to the stomach) or trauma to other parts of the body. Acute respiratory distress syndrome is life-threatening.

At the time of designation respiratory distress syndrome affected 0.5 to 2.3 in 10,000 persons in 10,000 people in the European Union (EU) *. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 19,000 to 87,000 people.

* Disclaimer: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001) and may differ from the true number of patients affected by the condition.

No medicinal products were authorised for the treatment of acute respiratory distress syndrome in the Community at the time of submission of the application for orphan drug designation. The treatment options for a majority of acute respiratory distress syndrome patients were limited to symptomatic care like oxygen treatment. Antibiotics and steroids may also be used to treat infections and reduce inflammation.

Lung surfactant is a protein and fat complex that coats the alveoli of the lung. It reduces surface tension and keeps the alveoli open, thus ensuring a stable surface for oxygen passage. Lusupultide is a human-like lung surfactant and, according to the sponsor, lusupultide ultimately will help the patient to breath better.

The effects of lusupultide were evaluated in experimental models. At the time of submission of the application for orphan designation, two clinical trials in patients with acute respiratory distress syndrome were completed.
Lusupultide was not marketed anywhere worldwide for the treatment of acute respiratory distress syndrome, at the time of submission. Orphan designation of lusupultide was granted in the United States for the treatment of adult respiratory distress syndrome
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 21 November 2000 a positive opinion recommending the grant of the above-mentioned designation.

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.