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.
The medicinal product has been authorised in the EU as Roctavian since 24 August 2022.
Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene
Treatment of haemophilia A
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Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Roctavian at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. More information is available in the orphan medicine assessment report.
Documents related to this orphan designation evaluation
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
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.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: