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Orphan designation

On 5 March 2012, orphan designation (EU/3/12/968) was granted by the European Commission to Envestia Limited, United Kingdom, for human monoclonal antibody targeting Staphylococcus aureus alpha-toxin (also known as KBSA301) for the treatment of pneumonia caused by Staphylococcus aureus.

The sponsorship was transferred to Global Regulatory Limited, United Kingdom, in November 2017.

What is 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.

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

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).

What treatments are available?

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.

How is this medicine expected to work?

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.

What is the stage of development of this medicine?

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.

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.

Key facts

Product details for <p>Human monoclonal antibody targeting Staphylococcus aureus alpha-toxin</p>
Active substanceHuman monoclonal antibody targeting Staphylococcus aureus alpha-toxin
Medicine Name
Disease/conditionTreatment of pneumonia caused by Staphylococcus aureus
Date of decision05/03/2012
Orphan decision numberEU/3/12/968

Review of designation

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

Sponsor’s contact details:

Global Regulatory Limited
266 - 268 Wickham Road
Croydon CR0 8BJ
United Kingdom
Tel: +44 20 86540555

Patients' organisations:

For contact details of patients’ organisations whose activities are targeted at rare diseases see:

  • Orphanet, a database containing information on rare diseases which includes a directory of patients’ organisations registered in Europe.
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.