EU/3/14/1357

On 19 November 2014, orphan designation (EU/3/14/1357) was granted by the European Commission to Numedicus Ltd, United Kingdom, for imatinib for the treatment of acute respiratory distress syndrome.

The sponsorship was transferred to Exvastat - United Kingdom in March 2017.

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 failure to deliver enough oxygen to 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, 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. 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 problems with breathing.

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

At the time of designation, acute respiratory distress syndrome affected less than 5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 256,000 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 511,100,000 (Eurostat 2014).

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 (substance similar to the one lining the airways that allows the lungs to expand properly) although they were not authorised for the condition.

How is this medicine expected to work?

Imatinib is a protein-tyrosine-kinase inhibitor. This means that it blocks some enzymes known as tyrosine kinases. In acute respiratory distress syndrome, imatinib is expected to work through several mechanisms, including by blocking a tyrosine kinase known as ARG (Abl-related gene). Early studies have shown that imatinib’s blocking of ARG helps keep the barrier between the air sacs and the blood intact, suggesting that the medicine could reduce the amount of fluids entering air sacs and thereby reduce the symptoms of the disease.

What is the stage of development of this medicine?

The effects of imatinib have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with imatinib in patients with acute respiratory distress syndrome had been started.

At the time of submission, imatinib was authorised in the EU for the treatment of several types of cancer including chronic myeloid leukaemia.

At the time of submission, imatinib 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 9 October 2014 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.

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.