EU/3/08/579: Orphan designation for the corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns

Ex-vivo-expanded autologous human corneal epithelium-containing stem cells

Overview

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.

Ex vivo expanded autologous human corneal epithelium containing stem cells has been authorised in the EU as Holoclar since 17 February 2015.

The sponsorship was transferred to Holostem Terapie Avanzate S.r.l., Italy in June 2020.

Key facts

Active substance
Ex-vivo-expanded autologous human corneal epithelium-containing stem cells
Medicine name
Holoclar
Intended use
Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns
Orphan designation status
Positive
EU designation number
EU/3/08/579
Date of designation
07/11/2008
Sponsor

Holostem Terapie Avanzate S.r.l. 
Via Glauco Gottardi 100 
Modena 
MO 
41125 
Italy 
Tel. +39 349 2403930
E-mail: sescretariat@holostem.com

Review of designation

During its meeting of 7 to 9 January 2015, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/08/579 for Holoclar (ex vivo expanded autologous human corneal epithelium containing stem cells) as an orphan medicinal product for the treatment of corneal lesions, with associated corneal (limbal) stem-cell deficiency, due to ocular burns. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment exist in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with this condition. The COMP recommended that the orphan designation of the medicine be maintained1.


1The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Holoclar (ex vivo expanded autologous human corneal epithelium containing stem cells) for the treatment of corneal lesions, wit... (PDF/84.72 KB)


    First published: 17/03/2015
    Last updated: 17/03/2015
    EMA/15233/2015

  • Patients' organisations

    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

    • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

    • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

    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:

    Related content

    How useful was this page?

    Add your rating
    Average
    1 rating