EU/3/05/331 - orphan designation for treatment of malabsorption due to exocrine pancreatic enzyme insufficiency

recombinant microbial lipase

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in June 2012 on request of the Sponsor.

On 28 October 2005, orphan designation (EU/3/05/331) was granted by the European Commission to Solvay Pharmaceuticals GmbH, Germany, for recombinant microbial lipase for the treatment of malabsorption due to exocrine pancreatic enzyme insufficiency.

Solvay Pharmaceuticals GmbH changed name to Abbott Products GmbH in November 2010.

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 (that 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 is the estimated number of patients affected by the condition?

At the time of designation, malabsorption due to exocrine pancreatic enzyme insufficiency affected approximately 3.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 177,000 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 466,600,000 (Eurostat 2005).

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 reduce the amounts of drug substance needed by the patients and thus improve the patient's ability to follow the required intake. 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 an artificially produced 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 medicine?

The evaluation of the effects of recombinant microbial 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 insufficie were initiated.

Recombinant microbial lipase was not authorised anywhere worldwide for the treatment of malabsorption due to exocrine pancreatic enzyme insufficiency or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 September 2005 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/3/05/331: Public summary of positive opinion for orphan designation of recombinant microbial lipase for the treatment of malabsorption due to exocrine pancreatic enzyme insufficiency

AdoptedReference Number: EMEA/COMP/315315/2005 Rev. 2

Key facts

• Active substance:
  recombinant microbial lipase
• Intended use:
  Treatment of malabsorption due to exocrine pancreatic enzyme insufficiency
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/05/331
• Date of designation:
  28/10/2005
• Sponsor:

  Abbott Products GmbH; Hans-Böckler-Allee 20; 30173 Hannover; Germany; Tel. [phone]; Fax [phone]