EU/3/04/222

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Orphan designation

On 2 September 2004, orphan designation (EU/3/04/222) was granted by the European Commission to Dr Falk Pharma GmbH, Germany, for pancreatic enzymes (cross linked enzyme crystal lipse, protease, amylase) for the treatment of malabsorption due to exocrine pancreatic enzyme insufficiency.

The sponsorship was transferred to Gregory Fryer Associates Ltd, United Kingdom, in June 2008 and subsequently to Eli Lilly Nederland B.V., The Netherlands, in May 2011.

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

At the time of designation, malabsorption due to exocrine pancreatic enzyme insufficiency affected approximately 3.6 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 166,000 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 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.

What treatments are 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 have better efficacy than other available enzymes. 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?

The proposed medicinal product contains the following 3 enzymes: crystal lipase, protease and amylase. By administration of this product it is expected that it will substitute the lacking pancreatic enzymes and ultimately restore normal digestion.

What is the stage of development of this medicine?

The effects of pancreatic enzymes (cross linked enzyme crystal lipase, protease, amylase) were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with malabsorption due to exocrine pancreatic insufficiency were ongoing.

Pancreatic enzymes (cross linked enzyme crystal lipase, protease, amylase) were not marketed anywhere worldwide for malabsorption due to exocrine pancreatic insufficiency, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 22 July 2004 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.

Key facts

Product details for <p>Pancreatic enzymes (cross linked enzyme crystal lipase, protease, amylase)</p>
Active substancePancreatic enzymes (cross linked enzyme crystal lipase, protease, amylase)
Medicine Name
Disease/conditionTreatment of malabsorption due to exocrine pancreatic enzyme insufficiency
Date of decision02/09/2004
OutcomePositive
Orphan decision numberEU/3/04/222

Review of designation

Sponsor’s contact details

Eli Lilly Nederland B.V. 
Grootslag 1-5 
NL-3991 RA Houten 
The Netherlands
Tel. +31 3060 25800

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.