EU/3/13/1178 - orphan designation for treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis

pegylated recombinant anti-Pseudomonas aeruginosa antibody

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in January 2015 on request of the sponsor.

On 5 August 2013, orphan designation (EU/3/13/1178) was granted by the European Commission to KaloBios Ltd, United Kingdom, for pegylated recombinant anti-Pseudomonas aeruginosa PcrV Fab' antibody for the treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis.

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

What is Pseudomonas aeruginosa lung infection in cystic fibrosis?

Cystic fibrosis is a genetic disease that affects the production of secretions such as mucus in the body. In patients with cystic fibrosis, there is an overproduction of thick mucus in the lungs, which leads to inflammation and a high risk of the lungs becoming infected with bacteria. Pseudomonas aeruginosa (P. aeruginosa) is one of the most common types of bacteria that tend to infect the lungs of patients with cystic fibrosis.

P. aeruginosa lung infection in cystic fibrosis is a long-term debilitating disease and may be life threatening because it severely damages the lung tissue and does not allow the patient to breathe normally.

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

At the time of designation, P. aeruginosa lung infection in cystic fibrosis affected less than 0.6 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 31,000 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).

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* 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 28), Norway, Iceland and Liechtenstein.
At the time of designation, this represented a population of 512,200,000 (Eurostat 2013).

What treatments are available?

At the time of designation, P. aeruginosa lung infection in patients with cystic fibrosis was mainly treated with antibiotics (medicines that kill bacteria). These were available as tablets, infusions (drips into a vein) and solutions for inhalation using a special machine called a nebuliser that changes the solution into an aerosol so that the patient can breathe it in.

Other medicines used to treat the lung symptoms of cystic fibrosis included bronchodilators that help open up the airways in the lungs and mucolytics that help to dissolve the mucus.

The sponsor has provided sufficient information to show that pegylated recombinant anti-P. aeruginosa PcrV Fab' antibody might be of significant benefit for patients with P. aeruginosa lung infection in cystic fibrosis because it works in a different way to existing treatments, by targeting the protein that enables P. aeruginosa to attack and kill the host cell. Early studies show that it may reduce infection and inflammation of the airways, including when used together with existing antibiotic treatments. 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?

The medicine consists of an antibody called the 'anti-P. aeruginosa PcrV Fab' antibody, which blocks the action of a protein called PcrV. This protein is used by the P. aeruginosa bacterium to attack and kill human cells, either by puncturing them or injecting toxins into them. By blocking this protein, the medicine is expected to prevent the effects of infection with P. aeruginosa in patients with cystic fibrosis.

The antibody is produced by a method known as 'recombinant DNA technology': it is made by cells into which genes (DNA) have been introduced that makes the cells able to produce the antibody. The antibody has also been modified by a process called 'pegylation'. This means that a chemical called 'polyethylene glycol' has been attached to the antibody, which is expected to prolong the time needed to remove the medicine from the body.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in cystic fibrosis patients with P. aeruginosa lung infection were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for P. aeruginosa lung infection in patients with cystic fibrosis 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 July 2013 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.

EU/3/13/1178: Public summary of opinion on orphan designation: Pegylated recombinant anti-Pseudomonas aeruginosa PcrV Fab’ antibody for the treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis

Adopted Reference Number: EMA/COMP/440852/2013 Rev.1

Key facts

• Active substance:
  pegylated recombinant anti-Pseudomonas aeruginosa antibody
• Intended use:
  Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/13/1178
• Date of designation:
  05/08/2013
• Sponsor:

  KaloBios Ltd; 5 Fleet Place; London; EC4M 7RD; United Kingdom; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

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