EU/3/01/076 - orphan designation for prevention of Pseudomonas aeruginosa infections in patients with cystic fibrosis

Octavalent Pseudomonas aeruginosa O-polysaccharide-toxin A conjugate

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in September 2006 on request of the sponsor.

On 11 December 2001, orphan designation (EU/3/01/076) was granted by the European Commission to Orphan Europe SARL, France, for octovalent Pseudomonas aeruginosa O-polysaccharide-toxin A conjugate vaccine for the prevention of Pseudomonas aeruginosa infections in patients with cystic fibrosis.

What is Pseudomonas aeruginosa infection in patients with cystic fibrosis?

Chronic infection of the lung with the bacterium Pseudomonas aeruginosa represents a hallmark of cystic fibrosis. It can induce damage to the lung tissue and respiratory insufficiency, which is life threatening.

What is the estimated number of patients at risk of developing the condition*?

At the time of designation Pseudomonas aeruginosa infection in patients with cystic fibrosis affected approximately 1.5 in 10,000 people in the European Union (EU)*. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 56,000 people.

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union. This represents a population of 377,000,000 (Eurostat 2001).

What methods of prevention are available?

There were no medicinal products authorised for the prevention of Pseudomonas aeruginosa infections in patients with cystic fibrosis in the Community, at the time of submission of the application for orphan designation.

How is this medicine expected to work?

Octovalent Pseudomonas aeruginosa O-polysaccharide-toxin A conjugate vaccine consists of specific parts from the Pseudomonas aeruginosa bacterium. The vaccine is given to the patient with the aim to activate the patient's immune system against any Pseudomonas aeruginosa, and prepare the body to fight larger amounts of the bacteria.

What is the stage of development of this medicine?

The effects of octovalent Pseudomonas aeruginosa O-polysaccharide-toxin A conjugate vaccine were evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in patients with cystic fibrosis were ongoing.

Octovalent Pseudomonas aeruginosa O-polysaccharide-toxin A conjugate vaccine was not marketed anywhere worldwide for the prevention of Pseudomonas aeruginosa infections in patients with cystic fibrosis or 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 26 October 2001 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.

EU/3/01/076: Public summary of positive opinion for orphan designation of octovalent Pseudomonas aeruginosa O-polysaccharide-toxin A conjugate vaccine for the prevention of Pseudomonas aeruginosa infections in patients w...

Adopted Reference Number: EMEA/COMP/86914/2009

Key facts

• Active substance:
  Octavalent Pseudomonas aeruginosa O-polysaccharide-toxin A conjugate
• Intended use:
  Prevention of Pseudomonas aeruginosa infections in patients with cystic fibrosis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/01/076
• Date of designation:
  11/12/2001
• Sponsor:

  Orphan Europe SARL; Immeuble Le Wilson; 70 Avenue du Général de Gaulle; F-92800 Puteaux; France; Tel. [phone]; E-mail: [e-mail]