EU/3/17/1849

About

On 20 March 2017, orphan designation (EU/3/17/1849) was granted by the European Commission to Freeline Therapeutics Ltd, United Kingdom, for adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene for treatment of Fabry disease.

The sponsor's address was updated in July 2018.

The sponsorship was transferred to Freeline Therapeutics (Ireland) Limited Ireland in May 2019.

Key facts

Active substance
Adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene
Disease / condition
Treatment of Fabry disease
Date of first decision
20/03/2017
Outcome
Positive
EU designation number
EU/3/17/1849

Review of designation

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

Sponsor's contact details

Freeline Therapeutics (Ireland) Limited
6th Floor, 2 Grand Canal Square
Dublin 2
Ireland
E-mail: contact@freeline.life

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;

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