EU/3/05/322 - orphan designation for treatment of acute peripheral arterial occlusion

alfimeprase

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in July 2008 on request of the sponsor.

On 28 October 2005, orphan designation (EU/3/05/322) was granted by the European Commission to QuadraMed Limited, United Kingdom, for alfimeprase for the treatment of acute peripheral arterial occlusion.

The sponsorship was transferred to Bayer HealthCare AG, Germany, in June 2006 and subsequently to QuadraMed Limited, United Kingdom, in September 2007.

What is acute peripheral arterial occlusion?

Acute peripheral arterial closure results in the sudden cessation of blood flow to an extremity of the body. The arteries are the vessels in the body that supply oxygenated blood to the tissues. The main cause of a sudden closure of arteries leading to the extremities (peripheral arteries) is due to the local accumulation of cells and material within the artery (thrombus) or because a clot has been brought to its site of lodgment by the blood current (embolism). Typically, the affected limb shows pallor, feels cold and becomes painful. The local nerves may become severely damaged, leading to paralysis (inability to move the limb). The lack of oxygen in the limb due to the obstruction of the artery might lead to gangrene (a loss of viable tissue). The disease is seriously debilitating and potentially life-threatening.

What are the methods of treatment available?

Various products were authorised for treatment of the condition in countries in the Community at the time of submission of the application for orphan drug designation. Treatment consists of medications such as blood-thinners or medicinal products which can dissolve the blood clot. In addition, surgical interventions might be needed to restore the blood perfussion as soon as possible. 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 acute peripheral vascular occlusion because it may offer an additional way to dissolve the blood clot and thereby might improve the long-term outcome of the patients. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

What is the estimated number of patients affected by the condition*?

Based on the information provided by the sponsor and previous knowledge of the Committee, acute peripheral arterial occlusion was considered to affect approximately 1.5 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 69,000 persons.

* 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.

How is this medicinal product expected to act?

Enzymes are proteins produced by the human body that speed up the conversion of certain substances into other substances. Alfimeprase is a genetically modified (artificially produced) enzyme expected to dissolve the blood clot by direct action on fibrin, an insoluble protein formed during the normal clotting of the blood. This might lead to the dissolution of a solid blood clot.

What is the stage of development of this medicinal product?

The effects of alfimeprase have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with acute peripheral arterial occlusion were ongoing.
Alfimeprase was not authorised anywhere worldwide for treatment of acute peripheral arterial occlusion, at the time of submission. Orphan designation of alfimeprase was granted in United States for treatment of acute peripheral arterial occlusion.
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.

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 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.

EU/3/05/322: Public summary of positive opinion for orphan designation of alfimeprase for the treatment of acute peripheral arterial occlusion

Adopted Reference Number: EMEA/COMP/315314/2005 Rev.2

Key facts

• Active substance:
  alfimeprase
• Intended use:
  Treatment of acute peripheral arterial occlusion
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/05/322
• Date of designation:
  28/10/2005
• Sponsor:

  QuadraMed Limited; MG House; Rumbolds Hill; Midhurst; West Sussex, GU29 9BY; United Kingdom