EU/3/09/705

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Orphan designation

On 28 January 2010, orphan designation (EU/3/09/705) was granted by the European Commission to Envestia Limited, United Kingdom, for human monoclonal antibody against Pseudomonas aeruginosa IATS-O1 for the treatment of pneumonia caused by serotype O1 Pseudomonas aeruginosa.

What is pneumonia caused by serotype O1 Pseudomonas aeruginosa?

Pneumonia (infection of the lungs) can be caused by the bacterium Pseudomonas aeruginosa (P. aeruginosa). The bacterium is widespread in nature and is able to cause infections in people who are seriously ill, such as patients who are in intensive care (especially those who are unable to breathe by themselves) or people whose immune system (the body’s natural defences) is weakened. These patients often catch the disease while they are in hospital. The infection is difficult to cure because P. aeruginosa is resistant to many antibiotics.

Serotype O1 P. aeruginosa is a sub-group of bacteria that carry a specific type of lipopolysaccharide (a type of sugar) on their surface.

Pneumonia caused by serotype O1 P. aeruginosa is a long-term debilitating disease because it lengthens the time that patients spend in hospital. The disease may also be life threatening because the patients affected are often already very weak and have an increased mortality.

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

At the time of designation, pneumonia caused by serotype O1 P. aeruginosa affected approximately 1.8 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 91,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and 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 504,800,000 (Eurostat 2009).

What treatments are available?

At the time of submission of the application for orphan drug designation, several antibiotics were authorised in the EU to treat this condition. Patients with pneumonia that was suspected to be caused by P. aeruginosa were given treatment consisting of at least two antibiotics known to be effective against P. aeruginosa. These included carbapenems, cephalosporins, piperacillin with tazobactam and fluoroquinolones.

The sponsor has provided sufficient information to show that human monoclonal antibody against P. aeruginosa IATS-O1 might be of significant benefit for patients with pneumonia caused by serotype O1 P. aeruginosa 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?

Human monoclonal antibody against P. aeruginosa IATS-O1 is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to the lipopolysaccharide that is found on the surface of serotype O1 P. aeruginosa. Once the product is attached to the bacterium, it attracts phagocytes (specialised ‘scavenger’ cells of the immune system) that attack and dispose of the bacterium.

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 human monoclonal antibody against P. aeruginosa IATS-O1 in experimental models was ongoing.

At the time of submission, no clinical trials with the designated product in patients with pneumonia caused by serotype O1 P. aeruginosa had been started.

At the time of submission, human monoclonal antibody against P. aeruginosa IATS-O1 was not authorised anywhere in the EU for pneumonia caused by serotype O1 P. aeruginosa 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 5 November 2009 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 Community) 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 against <em>Pseudomonas aeruginosa IATS-O1</em></p>
Active substanceHuman monoclonal antibody against Pseudomonas aeruginosa IATS-O1
Medicine Name
Disease/conditionTreatment of pneumonia caused by serotype O1 Pseudomonas aeruginosa
Date of decision28/01/2010
OutcomePositive
Orphan decision numberEU/3/09/705

Review of designation

Sponsor’s contact details:

Envestia Limited
The Sanderum Centre
30A Upper High Street
Thame
Oxon OX9 3EX
United Kingdom
Telephone: +44 1844 210 690
Telefax: +44 1844 211 081
E-mail: info@envestia.com