LOVE, BY: Achille Mauro Porcheddu
- ENEA'S CASE -
The War Against One Rare Disease :
By Enea's Parents 2004 - 2012 -
Achille Mauro Porcheddu, Angela Maria Cossu
porchedduachillemauro@gmail.com
Mobile: +39 347 7865073
Sardinia - Italy
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HUMAN PLASMINOGEN
TREATMENT OF LIGNEOUS CONJUNCTIVITIS
POSITIVE OPINION FOR ORPHAN DESIGNATION by EMEA - European Medicines Agency
http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2009/11/human_orphan_000189.jsp&mid=WC0b01ac058001d12b
EMEA - European Medicines Agency
7 Westferry Circus, Canary Wharf, London, E14 4HB, UK
Tel. (44-20) 74 18 84 00 Fax (44-20) 75 23 70 40
E-mail: orphandrugs@emea.europa.eu
Web: http://www.emea.europa.euDocument Date: London, 16 January 2008
Doc.Ref.: EMEA/COMP/285792/2007EU/3/07/461:
Public summary
of positive opinion
for orphan designation of human plasminogen
for the treatment of ligneous conjunctivitis
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COMMITTEE FOR ORPHAN MEDICINAL PRODUCTSPUBLIC SUMMARY OFPOSITIVE OPINION FOR ORPHAN DESIGNATIONOFhuman plasminogenfor the treatment of ligneous conjunctivitis
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COMMITTEE FOR ORPHAN MEDICINAL PRODUCTSPUBLIC SUMMARY OFPOSITIVE OPINION FOR ORPHAN DESIGNATIONOFhuman plasminogenfor the treatment of ligneous conjunctivitis
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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.
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.
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.
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.
* 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.
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: (i)
the seriousness of the condition, (ii) the existence or not of alternative methods of diagnosis,
prevention or treatment and (iii) 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.
For more information:
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 (Service not running?):
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 (N)
Source:
http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2009/10/WC500006465.pdf
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| HUMAN PLASMINOGEN TREATMENT OF LIGNEOUS CONJUNCTIVITIS Language - Active Ingredient - Indication |
| English - Human plasminogen - Treatment of ligneous conjunctivitis |
| Bulgarian - Човешки плазминоген - Лечение на лигнеозен конюнктивит |
| Czech - Lidský plazminogen - Léčba konjuktivitidy lignea |
| Danish - Humant plasminogen - Behandling af conjunctivitis lignosa |
| Dutch - Humaan plasminogen - Behandeling van conjunctivitis lignosa |
| Estonian - Inimese plasminogeen - Ligneoosse sidekestapõletiku ravi |
| Finnish - Ihmisen plasminogeeni - Conjunctivitis lignosan hoitoon |
| French - Plasminogène humain - Traitement de la conjonctivite ligneuse |
| German - Humanes Plasminogen - Behandlung der Konjunktivitis lignosa |
| Greek - Ανθρωπινο πλασμινογονο - Θεραπεία Ξυλώδους Επιπεφυκίτιδας |
| Hungarian - Humán plazminogén - Conjunctivitis lignosa kezelése |
| Italian - Plasminogeno umano - Trattamento delle congiuntivite lignea |
| Latvian - Cilvēka plazminogēns - Fibroza konjunktivīta ārstēšana |
| Lithuanian - Žmogaus plazminogenas - Medžių žiedadulkių sukelto konjunktyvito gydymas |
| Maltese - Plasminogen uman - Kura tal-konġuntivite linjuża |
| Polish - Plazminogen ludzki - Leczenie zapalenia spojówek z tworzeniem pseudobłony |
| Portuguese - Plasminogénio humano - Tratamento da conjuntivite lenhosa |
| Romanian - Plasminogen uman - Tratamentul conjunctivitei lemnoase |
| Slovak - Ľudský plazminogén - Liečba ligneóznej konjunktivitίdy |
| Slovenian - Človeški plazminogen - Tretma lesastega vnetja očesnih veznic |
| Spanish - Plasminógeno humano - Tratamiento de la conjuntivitis leñosa |
| Swedish - Human plasminogen - Behandling av träartad konjunktivit |
| Norwegian - Human plasminogen - Behandling av conjunctivitis lignosa |
| Icelandic - Forplasmín úr mönnum -
Meðferð við trjákenndri tárubólgu (iigneous conjunctivitis) |
Source:
http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2009/10/WC500006465.pdf
.
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LOVE, BY: Achille Mauro Porcheddu
- ENEA'S CASE -
The War Against One Rare Disease :
By Enea's Parents 2004 - 2012 -
Achille Mauro Porcheddu, Angela Maria Cossu
porchedduachillemauro@gmail.com -
Tel. +39 347 7865073
Address: Sardinia - Italy
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LOVE, BY: Achille Mauro Porcheddu
- ENEA'S CASE -
The War Against One Rare Disease :
By Enea's Parents 2004 - 2012 -
Achille Mauro Porcheddu, Angela Maria Cossu
porchedduachillemauro@gmail.com -
Tel. +39 347 7865073
Address: Sardinia - Italy
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