Valoctocogene roxaparvovec

This medicine is authorised for use in the European Union.


Roctavian is a medicine for treating severe haemophilia A, an inherited bleeding disorder caused by the lack of a clotting protein known as factor VIII. It is used in adults who do not have inhibitors (antibodies) against factor VIII and who have no antibodies against adeno-associated virus serotype 5 (AAV5).    

Roctavian contains the active substance valoctocogene roxaparvovec and is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.

Haemophilia A is rare, and Roctavian was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 21 March 2016. Further information on the orphan designation can be found here:

This EPAR was last updated on 12/09/2023

Authorisation details

Product details
Agency product number
Active substance
valoctocogene roxaparvovec
International non-proprietary name (INN) or common name
Valoctocogene roxaparvovec
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

Conditional approvalConditional approval

This medicine received a conditional marketing authorisation. This was granted in the interest of public health because the medicine addresses an unmet medical need and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required. For more information, see Conditional marketing authorisation.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
BioMarin International Limited
Date of issue of marketing authorisation valid throughout the European Union
Contact address

County Cork
P43 R298

Product information

08/09/2023 Roctavian - EMEA/H/C/005830 - IB/0007

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Product information documents contain:

You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.

Pharmacotherapeutic group


Therapeutic indication

Treatment of severe haemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).

Assessment history

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