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.

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

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 (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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