EU/3/06/381

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Orphan designation

On 29 June 2006, orphan designation (EU/3/06/381) was granted by the European Commission to MDS Pharma Services GB Limited, United Kingdom, for human monoclonal antibody against Pseudomonas aeruginosa serotype O11 for the treatment of pneumonia caused by serotype O11 Pseudomonas aeruginosa.

The sponsorship was transferred to Voisin Consulting, France, in July 2007 and subsequently to Envestia Limited, United Kingdom, in September 2014.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is pneumonia caused by serotype O11 Pseudomonas aeruginosa?

Pneumonia is an inflammation (reaction of the immune system, the body’s natural defence system) in the lung tissue. It can be caused by several microorganisms such as different kinds of bacteria, virus and fungi. It can also be caused by chemicals that are breathed in. The inflammation leads to a diminished function of the lungs and common symptoms are associated to the decreased ability of the blood to take up oxygen from the air. Especially in the hospital setting, pneumonia can be caused by infection by the bacteria Pseudomonas auruginosa. A serotype is defined by the kind of antibodies (proteins of the immune system that bind to foreign molecules) that are induced by different strains of microorganisms, in this case the bacterium. Serotype O11 is one of the most common strains of Pseudomonas auruginosa. Pneumonia caused by serotype O11 Pseudomonas aeruginosa is a life-threatening condition.

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

At the time of designation, pneumonia caused by serotype O11 Pseudomonas aeruginosa affected less than 5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 234,000 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).


*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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 468,900,000 (Eurostat 2006).

What treatments are available?

Several types of antibiotics (drugs that kill bacteria) were authorised for the treatment of the condition in the Community at the time of submission of application for orphan drug designation. Most antibiotics work by disrupting the building of the bacterial cell wall and thus preventing the bacteria from growing and multiplying.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that human monoclonal antibody against Pseudomonas aeruginosa serotype O11 might be of potential significant benefit for the treatment of pneumonia caused by serotype O11 Pseudomonas aeruginosa, mainly because it has a new mechanism of action. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

A monoclonal antibody is a protein that recognises and binds to a specific structure on a molecule. In this case, human monoclonal antibody against Pseudomonas aeruginosa serotype O11 is designed to bind to the specific type of Pseudomonas aureginosa bacteria called serotype O11. The antibody can promote the killing and elimination of the bacteria with the help of the the immune system. The medicinal product is intended to be used in combination with antibiotics. It is expected that the mode of action of this monoclonal antibody is killing Pseudomonas aeruginosa bacteria independent from the microbiological sensitivity of antibiotics.

What is the stage of development of this medicine?

The effects of human monoclonal antibody against Pseudomonas aeruginosa serotype O11 were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with pneumonia were ongoing.

Human monoclonal antibody against Pseudomonas aeruginosa serotype O11 was not authorised anywhere worldwide for the treatment of pneumonia, nor designated as orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 16 May 2006 a positive opinion recommending the grant of the above-mentioned 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;
  • 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.

Key facts

Product details for <p>Human monoclonal antibody against <em>Pseudomonas aeruginosa</em> serotype O11</p>
Active substanceHuman monoclonal antibody against Pseudomonas aeruginosa serotype O11
Medicine Name
Disease/conditionTreatment of pneumonia caused by serotype O11 Pseudomonas aeruginosa
Date of decision29/06/2006
OutcomePositive
Orphan decision numberEU/3/06/381

Review of designation

Sponsor’s contact details

Envestia Limited
The Sanderum Centre
30A Upper High Street
Thame
Oxfordshire
OX9 3EX
United Kingdom
Tel. +44 (0)184 4210 690
Fax +44 (0)184 4211 081
E-mail: info@envestia.com

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.