EU/3/18/2079

About

On 26 October 2018, orphan designation (EU/3/18/2079) was granted by the European Commission to Spark Therapeutics Ireland Limited, Ireland, for recombinant adeno-associated viral vector containing a bioengineered capsid and a codon-optimised expression cassette to drive the expression of the SQ form of a B-domain deleted human coagulation factor VIII (also known as SPK-8011) for the treatment of haemophilia A.

The sponsor’s address was updated in April 2019.

Key facts

Active substance
Recombinant adeno-associated viral vector containing a bioengineered capsid and a codon-optimised expression cassette to drive the expression of the SQ form of a B-domain deleted human coagulation factor VIII
Disease / condition
Treatment of haemophilia A
Date of first decision
26/10/2018
Outcome
Positive
EU designation number
EU/3/18/2079

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

Spark Therapeutics Ireland Limited
6th Floor
2 Grand Canal Quay
Dublin 2
Co. Dublin
Ireland
Tel: +353 87 2865356
E-mail: info@sparktx.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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