EU/3/06/387

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Orphan designation

On 25 July 2006, orphan designation (EU/3/06/387) was granted by the European Commission to Morgan Lewis & Bockius, United Kingdom, for amikacin sulfate (liposomal) for the treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis.

The sponsorship was transferred to Transave Inhalation Biotherapeutics Limited, United Kingdom, in July 2008. Transave Inhalation Biotherapeutics Limited changed its name to Insmed Limited in October 2011.

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 hereditary (genetic) disease that affects the production of secretions (such as mucus) from the glands in the body. It affects the lungs and the digestive system (gut) in particular. Cystic fibrosis is caused by abnormalities in a gene called ‘cystic fibrosis transmembrane conductance regulator’ (CFTR). The CFTR gene is responsible for the production of CFTR, a protein that regulates the production of mucus and digestive juices by acting as a chloride ion channel to allow proper movement of salt and water in and out of certain cells in the lungs and other tissues. In patients with cystic fibrosis, there is an overproduction of mucus in the lungs and a reduced production of digestive juices from the pancreas (an organ near the stomach). This leads to long-term infection and inflammation of the lungs and problems with the digestion and absorption of food resulting in poor growth. One of the most common kinds of bacterial infections is with Pseudomonas aeruginosa. In the long term, these events can induce damage to the lung tissue and the disease becomes life-threatening.

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

At the time of designation cystic fibrosis affected approximately 1.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 61,000 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 468,900,000 (Eurostat 2006).

What treatments are available?

At the time of submission of the application for orphan drug designation, lung infection and inflammation in cystic fibrosis were treated mainly with antibiotics (drugs that kill bacteria). These can be taken in a number of ways; as tablets, as intravenous infusion or as inhalation. Other medications to treat the lung symptoms of cystic fibrosis included bronchodilators (medications that enlarge the width of the airways) and mucolytics (drugs that help to dissolve the lung secretions). Associated treatments included daily exercise and physiotherapy and several other types of medications such as pancreatic enzymes and food supplements for the digestive symptoms. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that amikacin sulfate (liposomal) might be of potential significant benefit for the treatment of cystic fibrosis mainly because it may provide a major contribution to patient care. 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?

The medicinal product is an aminoglycoside, a kind of antibiotic that works by disrupting the building of the bacterial cell wall which consequently stops the bacteria from growing and multiplying in the lungs. This will prevent P. aeruginosa infection from developing in the lungs and prevent the inflammation that the infection causes.

What is the stage of development of this medicine?

The effects of amikacin sulfate (liposomal) 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.

Amikacin sulfate was authorised in several countries worldwide as an antibiotic, at the time of submission. Orphan designation of amikacin sulfate (liposomal) was granted in the United States for treatment of bronchopulmonary Pseudomonas aeruginosa infection in cystic fibrosis patients.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 15 June 2006 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>Amikacin sulfate (liposomal)</p>
Active substanceAmikacin sulfate (liposomal)
Medicine Name
Disease/conditionTreatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Date of decision25/07/2006
OutcomePositive
Orphan decision numberEU/3/06/387

Review of designation

Sponsor’s contact details

Insmed Limited
Overseas House
66-68 High Road
Bushey Heath
Hertfordshire WD23 1GG
United Kingdom
Tel. +44 (0)20 3114 5208
Fax +44 (0)20 8421 9883

Patients’ associations contact points:

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.