On 8 June 2004, orphan designation (EU/3/04/201) was granted by the European Commission to Ark Therapeutics Limited, United Kingdom, for vascular endothelial growth factor-D gene in an adenoviral vector for use with a collagen collar for the prevention of stenosis in synthetic grafts used in haemodialysis.
- What is stenosis in synthetic grafts used in haemodialysis?
In haemodialysis, the blood is allowed to flow through a machine with a special filter that removes wastes and extra fluids. The clean blood is then returned to the body. Removing the harmful wastes, extra salt and fluids helps control the blood pressure and keeps the proper balance of chemicals, like potassium and sodium, in the body. One important step before starting haemodialysis is preparing a vascular access, a site on the body from which blood is removed and returned.
A synthetic graft is a synthetic tube implanted under the skin of the arm, which connects one artery to one vein. The tube becomes a sort of artificial “robust” vein that can be used repeatedly for needle placement and blood access during haemodialysis. Such grafts are used in a minority of patients with chronic kidney failure and are meant to give a reliable access to haemodialysis. However, narrowing (stenosis) and loss of patency (the “openness”) at the vessel-graft junction is a common complication associated with these synthetic grafts. This results in reduced dialysis efficiency.
Stenosis in synthetic grafts used in haemodialysis requires a revision of this graft and is associated with a negative impact on quality of life.
- What is the estimated number of patients at risk of developing the condition?
At the time of designation, haemodialysis affected approximately 0.4 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 20,252 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).
- What are the methods of prevention available?
No satisfactory methods exist that were authorised at the time of application for orphan drug designation.
- How is this medicine expected to work?
Vascular endothelial growth factor-D gene in an adenoviral vector for use with a collagen collar is a gene medicine injected in a degradable cuff, which is placed around the junction of the vessel and the synthetic graft. It acts by trying to prevent narrowing of the blood vessel, ensuring a good flow of blood through the vessel.
- What is the stage of development of this medicine?
At the time of submission of the application for orphan designation, clinical trials in patients with synthetic grafts used in haemodialysis were ongoing.
The medicinal product was not marketed anywhere worldwide for prevention of stenosis in synthetic grafts used in haemodialysis, 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.
- 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.
|Name||Language||First published||Last updated|
|EU/3/04/201: Public summary of positive opinion for orphan designation: Vascular endothelial growth factor-D gene in an adenoviral vector for use with a collagen collar for the prevention of stenosis in synthetic grafts used in haemodialysis||(English only)||05/12/2004||04/05/2012|
|Active substance||Vascular endothelial growth factor-D gene in an adenoviral vector for use with a collagen collar|
|Disease/condition||Prevention of stenosis in synthetic grafts used in haemodialysis|
|Date of decision||08/06/2004|
|Orphan decision number||EU/3/04/201|
Review of designation
Sponsor’s contact details:
Ark Therapeutics Limited
44-46 Whitfield Street
London W1T 2RJ
Telephone: +44 20 73 88 77 22
Telefax: +44 20 73 88 78 05
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.