EU/3/03/183 - orphan designation for treatment of Burkholderia cepacia lung infection in cystic fibrosis

Temocillin sodium

Overview

On 14 January 2004, orphan designation (EU/3/03/183) was granted by the European Commission to Belpharma N.V, Belgium, for temocillin sodium for the treatment of Burkholderia cepacia lung infection in cystic fibrosis.

The sponsorship was transferred to Eumedica N.V., Belgium, in May 2014.

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

What is Burkholderia cepacia lung infection in cystic fibrosis?

Cystic fibrosis is a genetic disease. The genetic information that determines the characteristics of each individual is carried by genes located on structures called chromosomes. In humans, each cell has 23 pairs of chromosomes. For each pair, one chromosome is inherited from the mother and the other from the father. Cystic fibrosis is caused by abnormalities of a specific gene, called CFTR, carried by the seventh pair of chromosomes. The CFTR gene is responsible for the production of a protein that regulates outflow of water and salts (like chloride) from cells that cover internal and external surfaces of the body, the so-called epithelial cells. Cystic fibrosis appears only when the CFTR is abnormal on both chromosomes of the seventh pair. The defective transport of water and salts due to the lack of the regulatory protein results in the thickening of the secretions in several organs (e.g. lungs, pancreas). This leads to chronic infection of the lungs and chronic inflammation (a response to the injury caused to the tissue). Burkholderia cepacia is a bacterium (micro-organism that can cause certain types of infections). Infection of the lung with Burkholderia cepacia is a severe condition that can complicate cystic fibrosis. It can damage the lung tissue and cause respiratory insufficiency (failure of the lungs to provide adequately oxygen to the cells of the body and to remove the excess of carbon dioxide from them), which is life-threatening.

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

At the time of designation, Burkholderia cepacia lung infection in cystic fibrosis affected approximately 1.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 70,000 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 464,200,000 (Eurostat 2004).

What treatments are available?

Burkholderia cepacia lung infection in cystic fibrosis is treated mostly with antibiotics (drugs that kill micro-organisms) (e.g. meropenem and ceftazidime). At the time of submission of the application for orphan designation, antibiotics had not been specifically authorised for this condition in the Community. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that temocillin sodium might be of potential significant benefit for the treatment of B. cepacia lung infection in cystic fibrosis, particularly in regards to the narrow spectrum of activity of the medicinal product. This means in fact, that the use of temocillin sodium would not increase the risk of appearance of antibiotic resistance among infectious bacteria. The assumption of benefit is yet to be validated and 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?

Bacteria have an external wall with vital functions. This wall is formed by a complex structure in which several different molecules should be produced and assembled to end in the final structure. Temocillin sodium belongs to the group of drugs called penicillins. Penicillins prevent the linkage of some of the structures of the bacterial wall, resulting in an abnormal wall, unable to perform the vital functions of the bacteria, that finally will lead to the death of the bacteria.

What is the stage of development of this medicine?

At the time of the submission of the application for orphan designation temocillin sodium was authorised in three Member States of the European Community for the treatment of conditions other that the applied for orphan designation.

No clinical trials in patients with Burkholderia cepacia lung infection in cystic fibrosis had been initiated.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 December 2003 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/3/03/183: Public summary of positive opinion for orphan designation of temocillin sodium for the treatment of Burkholderia cepacia lung infection in cystic fibrosis

AdoptedReference Number: EMA/COMP/42574/2004 Rev. 2

Key facts

• Active substance:
  Temocillin sodium
• Intended use:
  Treatment of Burkholderia cepacia lung infection in cystic fibrosis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/03/183
• Date of designation:
  14/01/2004
• Sponsor:

  Eumedica N.V.; Avenue Winston Churchill 67; 1180 Brussels; Belgium; Tel. [phone]; Fax [phone]; E-mail: [e-mail]