EU/3/07/461

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Orphan designation

On 3 August 2007, orphan designation (EU/3/07/461) was granted by the European Commission to Kedrion S.p.A., Italy, for human plasminogen for the treatment of ligneous conjunctivitis.

What is ligneous conjunctivitis?

Ligneous conjunctivitis is a rare chronic inflammation of the eyes, which is caused by plasminogen deficiency and results in the formation of layers of different materials on the surface of the eyes (pseudomembranes). Plasminogen is a protein that is present in the blood and in the other tissues, including the eye, and also plays a role in the coagulation process. Ligneous conjunctivitis is chronically debilitating due to the risk of corneal involvement and subsequent blindness; it can
occasionally be life-threatening due to associated lung disease. 

What are the methods of treatment available?

No satisfactory methods exist that were authorised at the time of application. Surgery may be necessary in certain cases, but the lesions usually recur after surgery. Therapy with plasminogen, while not authorised, has been described in medical journals in a few patients with ligneous conjunctivitis, with apparent good results.

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

According to the information provided by the sponsor, ligneous conjunctivitis was considered to affect less than 5,000 persons in the European Union.

* 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?

Human plasminogen is expected to replace the missing protein in the eye and prevent pseudomembrane formation. Plasminogen is converted to plasmin, which helps to heal the lesions that develop in these patients.

What is the stage of development of this medicinal product?

At the time of submission of the application for orphan designation, no studies in experimental models, or clinical trials in patients, had been initiated. Human plasminogen was not authorised anywhere worldwide for ligneous conjunctivitis, or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 27 June 2007 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.

Key facts

Product details for <p>Human plasminogen</p>
Active substanceHuman plasminogen
Medicine Name
Disease/conditionTreatment of ligneous conjunctivitis
Date of decision03/08/2007
OutcomePositive
Orphan decision numberEU/3/07/461

Review of designation

Sponsor’s contact details:

Kedrion S.p.A.
Loc. Ai Conti
Castelvecchio Pascoli
Barga (Lucca)
Italy
Telephone: +39 0583 19 69 842
Telefax: +39 0583 19 69 825
E-Mail: info@kedrion.com

Patients’ association contact point:

KERATOS: European association on ocular surface diseases and lachrymal dysfunctions
55 avenue de la République
93170 Bagnolet
France
Telephone : +33 9 54 09 76 88
E-mail : keratos_contact@hotmail.com