Roctavian

RSS

Valoctocogene roxaparvovec

Authorised
This medicine is authorised for use in the European Union.

Overview

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: ema.europa.eu/medicines/human/orphan-designations/EU3161622.

This EPAR was last updated on 09/11/2022

Authorisation details

Product details
Name
Roctavian
Agency product number
EMEA/H/C/005830
Active substance
valoctocogene roxaparvovec
International non-proprietary name (INN) or common name
Valoctocogene roxaparvovec
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.

OrphanOrphan

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
Revision
1
Date of issue of marketing authorisation valid throughout the European Union
24/08/2022
Contact address

Shanbally
Ringaskiddy
County Cork
P43 R298
Ireland

Product information

09/11/2022 Roctavian - EMEA/H/C/005830 - IB/0002

This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.

 

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

Antihemorrhagics

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

Related content

How useful was this page?

Add your rating
Average
1 rating