EU/3/10/834

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Orphan designation

On 23 February 2011, orphan designation (EU/3/10/834) was granted by the European Commission to Talecris Biotherapeutics GmbH, Germany, for human plasmin for the treatment of acute peripheral arterial occlusion.

In January 2012, Talecris Biotherapeutics GmbH changed name to Grifols Deutschland GmbH.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

What is acute peripheral arterial occlusion?

Acute peripheral arterial occlusion is the sudden blockage of an artery supplying blood to the limbs by a blood clot. The blood clot can form locally because the artery is already narrowed, or it can come from another part of the body, for example after surgery to the heart. Typically, the patient experiences severe pain in the affected limb, which also becomes cold and pale. The local nerves may become severely damaged, leading to loss of sensation and the inability to move the limb. The lack of blood flow in the limb could also lead to tissue damage.

Acute peripheral arterial occlusion is a debilitating disease that may be life threatening, especially because of the irreversible tissue damage. It could also lead to amputation of the affected limb.

What is the estimated number of patients affected by acute peripheral arterial occlusion?

At the time of designation, acute peripheral arterial occlusion affected less than 1.7 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 86,000 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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 507,700,000 (Eurostat 2011).

What treatments are available?

At the time of designation, satisfactory methods had been authorised in the EU for the treatment of acute peripheral arterial occlusion. These included medicines that activate a protein called plasminogen into its active form, plasmin, which then breaks down the blood clots. In addition, surgery was used to restore the blood flow and prevent limb loss and disability.

The sponsor has provided sufficient information to show that human plasmin might be of significant benefit for patients with this condition because of its direct action on the blood clot causing the blockage of the arteries compared with the indirect action of existing treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Plasmin is a protein found naturally in the blood that breaks down blood clots. This medicine is a purified form of human plasmin, prepared from human plasma (the liquid part of the blood), which is expected to be delivered directly into the affected arteries of patients with acute peripheral arterial occlusion using a catheter (a thin sterile tube). When delivered, the medicine is expected to break down the clot, thereby removing the obstruction and relieving the patient’s symptoms.

What is the stage of development of this medicine?

The effects of human plasmin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with human plasmin in patients with acute peripheral arterial occlusion were ongoing.

At the time of submission, human plasmin was not authorised anywhere in the EU for acute peripheral arterial occlusion. Orphan designation of human plasmin had been granted in the United States of America for acute peripheral arterial occlusion.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 November 2010 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.

Key facts

Product details for <p>Human plasmin</p>
Active substanceHuman plasmin
Medicine Name
Disease/conditionTreatment of acute peripheral arterial occlusion
Date of decision23/02/2011
OutcomePositive
Orphan decision numberEU/3/10/834

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details

Grifols Deutschland GmbH
Lyoner Strasse 15
D-60528 Frankfurt am Main
Germany
Tel. +49 6966 0593 401
Fax +49 6966 0593 110
E-mail: wolfgang.schulten@talecris.com

Patients' organisations

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.