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Orphan designation

On 28 July 2015, orphan designation (EU/3/15/1511) was granted by the European Commission to ProMetic BioTherapeutics Ltd, United Kingdom, for human plasminogen for the treatment of plasminogen deficiency.

What is plasminogen deficiency?

Plasminogen deficiency is an inherited disorder in which patients cannot produce normal amounts of plasminogen or in which plasminogen does not work properly. Plasminogen is a protein important in dissolving blood clots and helping wounds to heal. The first type, which is the more common form in Europe, is usually more severe and is associated with the formation of pseudomembranes, fibrous layers of protein, on moist body surfaces such as the conjunctiva (the lining of the inner eyelids and outer layer of the white of the eye), the lining of the mouth and the linings of the windpipe, gut, and vagina. This can lead to inflammation and organ damage. In addition, some children with the condition are born with fluid in the brain which can lead to brain damage if untreated.

Plasminogen deficiency is a long-term debilitating condition due to damage to the eyes and other organs.

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

At the time of designation, plasminogen deficiency affected approximately 0.02 in 10,000 people in the European Union (EU). This was equivalent to a total of around 1,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 28), Norway, Iceland and Liechtenstein. This represents a population of 512,900,000 (Eurostat 2015).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for treating plasminogen deficiency. Patients were managed with surgery and with medicines to reduce inflammation and scarring.

How is this medicine expected to work?

This medicine contains purified plasminogen extracted from human blood. When given by injection to patients who lack working plasminogen of their own, it is expected to replace the missing protein in their blood and so correct the symptoms of the condition.

What is the stage of development of this medicine?

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

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with plasminogen deficiency were ongoing.

At the time of submission, human plasminogen was not authorised anywhere in the EU for plasminogen deficiency. Orphan designation of the medicine had been granted in the United States for type 1 plasminogen deficiency (hypoplasminogenaemia).

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 June 2015 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 plasminogen</p>
Active substanceHuman plasminogen
Medicine Name
Disease/conditionTreatment of plasminogen deficiency
Date of decision28/07/2015
Orphan decision numberEU/3/15/1511

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

ProMetic BioTherapeutics Ltd
Horizon Park, Barton Road
Cambridge CB23 7AJ
United Kingdom
Tel. +44 (0)1223 433822
Fax +44 (0)1223 420270

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.