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Orphan designation

On 22 May 2006 orphan designation (EU/3/06/371) was granted by the European Commission to Ockham Biotech Limited, United Kingdom, for heparin sodium 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.
Heparin sodium might be of potential significant benefit for the treatment of cystic fibrosis because it might improve the long-term outcome of patients. 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?

Heparin sodium is a product mainly known by its ability to dissolve blood clots. The sponsor of this product is developing a formulation of heparin sodium that could be taken by inhalation. In cystic fibrosis, the product is thought to dissolve the mucus and to decrease inflammation. It is expected that using heparin sodium locally in the airways the product might help reducing the symptoms of the disease.

What is the stage of development of this medicine?

The effects of heparin sodium were evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with treatment of cystic fibrosis were ongoing.
Heparin sodium was not authorised anywhere worldwide for treatment of cystic fibrosis, at the time of submission. Orphan designation of heparin sodium was granted in the United Kingdom for treatment of cystic fibrosis.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 5 April 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;
  • 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.

Key facts

Product details for <p>Heparin sodium</p>
Active substanceHeparin sodium
Medicine Name
Disease/conditionTreatment of cystic fibrosis
Date of decision22/05/2006
Orphan decision numberEU/3/06/371

Review of designation

Sponsor’s contact details:

Ockham Biotech Limited
Manor Farm
98 Swanwick Lane
Hampshire SO31 7HA
United Kingdom
Telephone: +44 2392 84 21 52
Telefax: +44 2392 84 35 65

Patients’ associations contact points:

Cystische Firbose Hilfe Österreich
Hanuschgasse 1
2540 Bad Vöslau
Telephone: +43 2252 89 00 18
Telefax: +43 2252 89 00 18 15

Vaincre la Mucoviscidose
181, rue de Tolbiac
75013 Paris
Telephone: +33 1 40 78 91 91
Telefax: +33 1 45 80 86 44

Cystic Fibrosis Trust
11 London Road
United Kingdom
Telephone: +44 20 84 64 72 11
Telefax: +44 20 83 13 04 72