On 6 March 2002, orphan designation (EU/3/02/086) was granted by the European Commission to Axcan Pharma International BV, the Netherlands, for porfimer sodium (for use with photodynamic therapy) for the treatment of high-grade dysplasia in Barrett’s oesophagus.
The sponsorship was transferred to Pinnacle Biologics B.V., The Netherlands, in August 2011.
- What is high-grade dysplasia in Barrett’s oesophagus?
Barrett’s oesophagus (BO) is a condition in which the normal lining of the lower part of the gullet, is replaced over time by another type of lining, normally present in the stomach. BO is clearly recognisable at endoscopy. Usually BO develops during the process of healing after a chronic injury to the gullet, such as caused by reflux of gastric juice from the stomach to the gullet. Continued reflux causes initially mild (low-grade), and later, severe (high-grade) dysplasia. Dysplasia may lead to cancer of the gullet (oesophageal carcinoma) and as such is a life-threatening condition.
- What is the estimated number of patients affected by the condition?
At the time of designation, high-grade dysplasia in Barrett’s oesophagus affected approximately 3.6 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 136,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).
*Disclaimer: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001) and may differ from the true number of patients affected by the condition. This estimate is based on available information and calculations presented by the sponsor at the time of the application.
- What treatments are available?
High-grade dysplasia requires careful endoscopic follow-up with multiple biopsies. As an alternative approach, surgery of the BO segment can be used. At the time of submission of the application for orphan designation, no satisfactory method had been authorised in the European Union for treatment of the condition.
- How is this medicine expected to work?
Porfimer sodium is a photosensitizing agent used in photodynamic therapy. The actions of porfimer sodium are light and oxygen dependent. The induced cell toxicity may be due to free radical generation and through interruption of the blood supply to the tumour.
- What is the stage of development of this medicine?
The effects of the medicinal product have been evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in patients with high-grade dysplasia in BO were ongoing.
Porfimer sodium had not been marketed anywhere worldwide for high grade dysplasia in BO or designated as an 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 18 December 2001 recommending the granting of this designation.
- Opinions on orphan medicinal products designations are based on the following cumulative criteria:
- the seriousness of the condition,
- the existence or not 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.
|Name||Language||First published||Last updated|
|EU/3/02/086: Public summary of positive opinion for orphan designation of porfimer sodium (for use with photodynamic therapy) for the treatment of high-grade dysplasia in Barrett’s oesophagus||(English only)||27/02/2007||11/11/2011|
|Active substance||Porfimer sodium (for use with photodynamic therapy)|
|Disease/condition||Treatment of high-grade dysplasia in Barrett's oesophagus|
|Date of decision||06/03/2002|
|Orphan decision number||EU/3/02/086|
Review of designation
Sponsor’s contact details:
Pinnacle Biologics B.V.
p/a Trust Company Amsterdam B.V.
Crystal Tower 21st Floor
1043 DP Amsterdam
Telephone: +31 20 386 86 22
Telefax: +31 20 203 11 96
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.