EU/3/05/323 - orphan designation for treatment of malabsorption due to exocrine pancreatic enzyme insufficiency
bacterial lipase (burlulipase)
OrphanHuman
Overview
This medicine is now known as burlulipase.
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.
The sponsorship was transferred to Nordmark Pharma GmbH in June 2021.
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.
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.
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.
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.
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.
- 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
- Active substance
- bacterial lipase (burlulipase)
- Intended use
- Treatment of malabsorption due to exocrine pancreatic enzyme insufficiency
- Orphan designation status
- Positive
- EU designation number
- EU/3/05/323
- Date of designation
- Sponsor
Nordmark Pharma GmbH
Pinnauallee 4
25436 Uetersen
Schleswig-Holstein
Germany
E-mail: info@nordmark-pharma.de
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: