EU/3/17/1835: Orphan designation for the treatment of epidermolysis bullosa

Ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene (prademagene zamikeracel)

Overview

On 27 February 2017, orphan designation (EU/3/17/1835) was granted by the European Commission to Ser-mes Planificación SL, Spain, for ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene (also known as EB-101) for the treatment of epidermolysis bullosa.

Key facts

Active substance
Ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene (prademagene zamikeracel)
Intended use
Treatment of epidermolysis bullosa
Orphan designation status
Positive
EU designation number
EU/3/17/1835
Date of designation
27/02/2017
Sponsor

Pharma Gateway AB
Johanneslundsvägen 2
Hammarby
194 61 Upplands Väsby
Stockholms Lan
Sweden
Tel. +46 8 590 778 00
E-mail: info@pharmagateway.eu

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product it it is approved for marketing authorisation.

Update history

DateUpdate
January 2023The sponsorship was transferred from Abeona Therapeutics Europe S.L., Spain to Pharma Gateway AB, Sweden.

Documents related to this orphan designation evaluation

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:

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