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.
EU/3/16/1622: Public summary of opinion on orphan designation: Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene for the treatment of haemophilia A (PDF/126.17 KB)
First published: 04/05/2016
Last updated: 04/05/2016
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||
|EU designation number||
Review of designation
Sponsor's contact details
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.