EU/3/05/323

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Orphan designation

On 7 November 2005, orphan designation (EU/3/05/323) was granted by the European Commission to Nordmark Arzneimittel GmbH u. Co. KG, Germany, for bacterial lipase for the treatment of malabsorption due to exocrine pancreatic enzyme insufficiency.

What is malabsorption due to exocrine pancreatic enzyme insufficiency?

The pancreas is a small organ that lies behind the stomach and in front of the spine. The pancreas has two main functions in the body. It makes hormones, such as insulin, that help to control blood sugar levels (this is called endocrine activity). It also makes a juice that helps to digest (break down) food (this is called exocrine activity). The juice contains enzymes, which are proteins that speed up the transformation of certain substances (such as particles of food) into other substances.
Certain conditions affecting the pancreas, including cystic fibrosis, inflammation of the pancreas (pancreatitis), cancer of the pancreas and an inherited disease called Shwachman-Diamond syndrome, may interfere with its exocrine activity by reducing the synthesis and the release of pancreatic enzymes. This may result in a defective absorption (malabsorption) of nutrients. Typically, the patient experiences bloating and pain in the abdominal area and loose, frequent stools. Malabsorption due to exocrine pancreatic enzyme insufficiency is a severe chronically debilitating condition.

What are the methods of treatment available?

Several pancreatic enzyme extracts were authorised for treatment of the condition at the time of application. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that the medicinal product might be of potential significant benefit for the treatment of malabsorption due to exocrine pancreatic insufficiency because it may reduce the amounts of drug substance needed by the patients compared to the actual authorised drugs and thus improve the patient s ability to follow the required intake of drugs. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status. 

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

According to the information provided by the sponsor, malabsorption due to exocrine pancreatic enzyme insufficiency was considered to affect about 166,000 persons in the European Union.

*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 Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

The proposed medicinal product contains lipase, an enzyme that breaks down fat. By administration of this product it is expected that it will substitute the lacking pancreatic enzyme and improve the digestion. 

What is the stage of development of this medicinal product?

The evaluation of the effects of bacterial lipase in experimental models is ongoing. At the time of submission of the application for orphan designation, no clinical trials in patients with malabsorption due to exocrine pancreatic enzyme insufficiency were initiated. Bacterial lipase was not authorised anywhere worldwide for malabsorption due to exocrine pancreatic enzyme insufficiency 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 9 September 2005 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition,
  • the existence or not of alternative methods of diagnosis, prevention or treatment and
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments. 

Designated orphan medicinal products are still investigational products which were considered for 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 will be necessary before this product can be granted a marketing authorisation.

Key facts

Product details for <p>Bacterial lipase</p>
Active substanceBacterial lipase
Medicine Name
Disease/conditionTreatment of malabsorption due to exocrine pancreatic enzyme insufficiency
Date of decision06/11/2005
OutcomePositive
Orphan decision numberEU/3/05/323

Review of designation

Sponsor’s contact details:

Nordmark Arzneimittel GmbH u. Co. KG
Pinauallee 4
25436 Uetersen
Germany
Telephone: +49 41 22 71 20
Telefax: +49 41 22 71 22 20
E-mail: info@nordmark-pharma.de

Patients’ associations contact points:

CF : Cystic Fibrosis Association of Ireland
24 Lower Rathmines Road
Dublin 6
Dublin
Ireland
Telephone: +353 1 49 62 433
Telefax: +353 1 49 62 201
E-mail: info@cfireland.ie

Mukoviszidose e.V.
Deutsche Gesellschaft zur Bekämpfung der Mukoviszdose, gemeinnütziger Verein
Bendenweg 101
53121 Bonn
Germany
Telephone: +49 22 89 87 800
Telefax: +49 22 89 87 80 77
E-mail: info@mukoviszidose-ev.de

Vaincre la Mucoviscidose
181, rue de Tolbiac
75013 Paris
France
Telephone : +33 1 40 78 91 91
Telefax : +33 1 45 80 86 44
E-mail : info@vaincrelamuco.org