Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2017 on request of the sponsor.
On 8 June 2004, orphan designation (EU/3/04/200) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for pasireotide for the treatment of functional gastro-entero-pancreatic endocrine tumours.
This medicine is now known as pasireotide.
Functional gastro-entero-pancreatic endocrine tumours are tumours originating in the digestive tract structures. These tumours produce and release excessive amounts of substances called hormones and of other substances that are normally present in the body in very small amounts. Different symptoms can be associated to different functional gastro-entero-pancreatic endocrine tumours depending on the type of substance(s)/hormone(s) produced by the tumour. The most common type among these tumours, is the so-called carcinoid tumour. It contains cells that synthesise a substance called serotonin that, in excess, mainly produces facial flushing (transient redness of the face and neck) and brief periods of watery stools.
Functional gastro-entero-pancreatic endocrine tumours are chronically debilitating and life-threatening to the patient.
At the time of designation, functional gastro-entero-pancreatic endocrine tumours affected approximately 0.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 28,000 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).
The choice of treatment of functional gastro-entero-pancreatic endocrine tumours depends on several factors, including the type of tumour and the stage of the disease. Treatments may include surgery, immunotherapy (treatment of disease by stimulating the body's own defence system), hormonal therapy or chemotherapy (using drugs to kill cancer cells). Several products were authorised for the condition in the Community at the time of submission of the application for orphan drug designation.
Pasireotide could be of potential significant benefit for the treatment of functional gastro-entero-pancreatic endocrine tumours as it might improve the long-term outcome of the patients. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.
Hormones are substances secreted by specialised cells in the body. They are recognised by certain proteins, called hormone receptors, which are present on the surface of some cells. The hormones will lock on to these receptors, causing a biological effect on the activity of the cell. Gastro-entero-pancreatic endocrine tumours also have this kind of receptors on their cell surface, which might link pasireotide. In this way pasireotide will help to regulate the production and release of the substances produced by the tumour cells.
The effects of pasireotide were evaluated in experimental models.At the time of submission of the application for orphan designation, clinical trials in patients with functional gastro-entero-pancreatic endocrine tumours were ongoing.
The medicinal product was not marketed anywhere worldwide for functional gastro-entero-pancreatic endocrine tumours 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 14 April 2004 recommending the granting of this designation.
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.
Novartis Europharm Limited
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: