On 11 April 2006, orphan designation (EU/3/06/361) was granted by the European Commission to Mukoviszidose e.V., Germany, for glutathione for the treatment of cystic fibrosis.
- What is 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.
Cystic fibrosis is a long lasting and life-threatening disease.
- 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 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 60,000 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 (EU 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).
- What treatments are available?
At the time of submission of the application for orphan drug designation, lung infection and inflammation in cystic fibrosis were mainly treated with physiotherapy and antibiotics. Other medicines used to treat the lung disease included bronchodilators (medicines that help to open up the airways in the lungs) and mucolytics (medicines that help dissolve the mucus in the lungs). In addition, patients are often given other types of medicine such as pancreatic enzymes (substances that help to digest and absorb food) and food supplements. They are also advised to exercise and to undergo physiotherapy.
Satisfactory argumentation has been submitted to justify the assumption that the glutathione administered by inhalation could be of potential significant benefit for the treatment of cystic fibrosis because it could improve the long-term outcome of the condition. The 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?
Glutathione is a substance that can protect cells against toxins such as free radicals. It is usually present at very high concentration in the thin layer of fluid surrounding the cells of the lung (epithelial lining fluid). In patients with cystic fibrosis the levels of glutathione in the lung drop. This medicinal product is expected to increase the levels of glutathione. This is believed to reduce inflammation and help maintain normal composition of mucus. This way it is expected to relieve the symptoms of the patients.
- What is the stage of development of this medicine?
The effects of glutathione have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with cystic fibrosis were ongoing.
Glutathione was not authorised anywhere in the world for 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 8 March 2006 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;
- 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.
|Name||Language||First published||Last updated|
|EU/3/06/361: Public summary of positive opinion for orphan designation of glutathione for the treatment of cystic fibrosis||(English only)||15/05/2009|
|Disease/condition||Treatment of cystic fibrosis|
|Date of decision||11/04/2006|
|Orphan decision number||EU/3/06/361|
Review of designation
Sponsor’s contact details:
In den Dauen 6
Telephone: +49 22 89 87 800
Telefax: +49 22 89 87 80 77
Patients’ associations contact points:
Vaincre la Mucoviscidose
181, rue de Tolbiac
Telephone: +33 1 40 78 91 91
Telefax: +33 1 45 80 86 44
CFAI - Cystic Fibrosis Association of Ireland
24 Lower Rathmines Road,
Telephone: +353 1 496 2433
Local call: 1890 311 211
Telefax: +353 1 496 2201
Deutsche Gesellschaft zur Bekämpfung der Mukoviszdose, gemeinnütziger Verein
Telephone: +49 22 89 87 800
Telefax: +49 22 89 87 80 77