EU/3/08/579

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Orphan designation

On 7 November 2008, orphan designation (EU/3/08/579) was granted by the European Commission to Chiesi Farmaceutici S.P.A., Italy, for ex vivo expanded autologous human corneal epithelium containing stem cells for the treatment of corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns.

What are corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns?

Corneal lesions are areas of damage to the cornea, the transparent surface at the front of the eye in front of the pupil. The surface of the cornea is constantly being renewed and replaced by the production of new cells that are produced by ‘stem cells’ in the lower layers of the cornea, called the ‘basal limbal epithelium’. Ocular burns (burns to the eye) because of chemicals or heat can damage these stem cells. This can cause a deficiency (low number) of the stem cells, reducing the renewal and replacement of the surface of the cornea. This results in the cornea being repaired by different types of eye cell, which can make the cornea opaque and impair the patient’s vision.

This condition is considered to be debilitating because of loss of vision.

What treatments are available?

There are no authorised products for this condition in the Community, although surgery, such as transplanting a cornea from a donor who has recently died, can been used. Although this can restore the patient’s vision, failure of the cornea can occur at any time after the transplant.
Ex vivo expanded autologous human corneal epithelium containing stem cells could be of potential significant benefit for the treatment of corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns, because they might act differently from other methods. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

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

At the time of designation corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns affected approximately 0.3 in 10,000 people in the European Union (EU)*. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 151,000 people.

* 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 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

How is this medicine expected to work?

This product is made of a small sample of cells that are taken from an undamaged part of the basal limbal epithelium in the patient’s cornea. The cells are attached to a ‘glue’ made of chemically-modified fibrin and grown in the laboratory to produce a replacement surface (epithelium) for the cornea. The epithelium is then implanted into the patient’s damaged eye or eyes. The stem cells contained within the epithelium then help the cornea to regenerate, restoring the patient’s vision.

What is the stage of development of this medicine?

The effects of this product have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns were ongoing.

At the time of submission, this product was not authorised anywhere worldwide for this condition 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 a positive opinion on 10 September 2008 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 Community) 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>Ex vivo expanded autologous human corneal epithelium containing stem cells</p>
Active substanceEx vivo expanded autologous human corneal epithelium containing stem cells
Medicine Name
Disease/conditionCorneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns
Date of decision06/11/2008
OutcomePositive
Orphan decision numberEU/3/08/579

Review of designation

Sponsor’s contact details:

Chiesi Farmaceutici S.p.A
Via Palermo, 26/A
43100 Parma
Italy
Telephone: + 39 0521 2791
Telefax: + 39 0521 77 41 20
E-mail: info@chiesigroup.com

Patients’ associations contact points:

The Eyecare Trust
PO Box 804
Aylesbury
Buckinghamshire
HP20 9DF
United Kingdom
Telephone: +44 845 129 5001
Telefax: +44 845 129 5001
E-mail: info@eyecaretrust.org.uk

KERATOS: Association sur les Pathologies de la Surface Oculaire et les Dysfonctionnements Lacrymaux
55 avenue de la République
93170 Bagnolet
France
Telephone : +33 9 54 09 76 88
E-mail : keratos_contact@hotmail.com

KÓROS : Associazione per la Ricerca e la Prevenzione delle Malattie Oculari Infantili - ONLUS
Viale Amerigo Vespucci 1/C
30173 Mestre (VE)
Italy
Telephone : +39 041 26 68 784
Telefax : + 39 041 53 51 831
E-mail: segreteria@korosonlus.org