EU/3/04/222 - orphan designation for treatment of malabsorption due to exocrine pancreatic enzyme insufficiency

Pancreatic enzymes (cross linked enzyme crystal lipase, protease, amylase)

Overview

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 and finally to TRX Services Limited, United Kingdom, in February 2015.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

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 was equivalent to a total of around 167,000 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 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 464,200,000 (Eurostat 2004).

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.

EU/3/04/222: Public summary of positive opinion for orphan designation of pancreatic enzymes (cross linked enzyme crystal lipase, protease, amylase) for the treatment of malabsorption due to exocrine pancreatic enzyme ...

Adopted Reference Number: EMEA/COMP/77766/2004 Rev.3

Key facts

• Active substance:
  Pancreatic enzymes (cross linked enzyme crystal lipase, protease, amylase)
• Intended use:
  Treatment of malabsorption due to exocrine pancreatic enzyme insufficiency
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/04/222
• Date of designation:
  02/09/2004
• Sponsor:

  TRX Services Limited; Global House; 5A Sandy's Row; London E1 7HW; United Kingdom; Tel. [phone]; Fax [phone]