EU/3/07/461 - orphan designation for treatment of ligneous conjunctivitis
Human plasminogen
OrphanHuman
Overview
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.
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.
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.
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.
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.
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.
- 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
- Active substance
- Human plasminogen
- Intended use
- Treatment of ligneous conjunctivitis
- Orphan designation status
- Positive
- EU designation number
- EU/3/07/461
- Date of designation
- Sponsor
Kedrion S.p.A.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: