EU/3/16/1622

About

On 21 March 2016, orphan designation (EU/3/16/1622) was granted by the European Commission to BioMarin Europe Ltd., United Kingdom, for adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene (also called BMN 270) for the treatment of haemophilia A.

The sponsorship was transferred to BioMarin International Limited, Ireland in September 2018.

Key facts

Active substance
Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene
Disease / condition
Treatment of haemophilia A
Date of first decision
21/03/2016
Outcome
Positive
EU designation number
EU/3/16/1622

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

BioMarin International Limited
Shanbally
Ringaskiddy
County Cork P43 R298
Ireland
Tel. +353 1479430
E-mail: shanbally@bmrn.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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