EU/3/12/968: Orphan designation for the treatment of pneumonia caused by Staphylococcus aureus

Pneumonia (infection of the lungs) can be caused by the bacterium Staphylococcus aureus (S. aureus). About 30% of people carry this bacterium in their nose, but most of the time it does not cause any harm. People who are seriously ill and whose immune system (the body's natural defences) is weakened are particularly vulnerable to infection with S. aureus. Meticillin-resistant S. aureus (MRSA) is a strain of S. aureus that has become resistant to most antibiotics and is therefore difficult to treat.

Pneumonia caused by S. aureus is a life-threatening disease because it may lead to septicaemia (blood infection) and respiratory failure.

At the time of designation, pneumonia caused by S. aureus affected not more than 1.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 61,000 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).

*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 506,300,000 (Eurostat 2011).

At the time of designation, several antibiotics were authorised in the EU to treat this condition.

The sponsor has provided sufficient information to show that 'human monoclonal antibody targeting Staphylococcus aureus alpha-toxin' might be of significant benefit for patients with pneumonia caused by S. aureus because it works in a different way to existing treatments, and early studies in experimental models indicate that it might be used together with antibiotics to improve the treatment of patients with this condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to the S. aureus alpha-toxin, which is one of the toxic substances released by the bacterium during the infection. By attaching to alpha-toxin, this medicine is expected to reduce the ability of the bacterium to cause damage to the body's cells.

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.

At the time of submission, no clinical trials with the medicine in patients with pneumonia caused by S. aureus had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for pneumonia caused by S. aureus 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 11 January 2012 recommending the granting of this designation.

• 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.