EU/3/18/2080

About

On 26 October 2018, orphan designation (EU/3/18/2080) was granted by the European Commission to Freeline Therapeutics Ltd, United Kingdom, for recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant (also known as FLT180a) for the treatment of haemophilia B.

Key facts

Active substance
Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant
Disease / condition
Treatment of haemophilia B
Date of decision
26/10/2018
Outcome
Positive
Orphan decision number
EU/3/18/2080

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 Ltd
Bioscience Catalyst
Gunnels Wood Road
Stevenage
Herts SG1 2FX
United Kingdom
Tel. +44 (0)20 7794 4227
E-mail: contact@freelinetx.com

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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