Roctavian
What Roctavian is used for
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).
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Advanced therapy: a gene, cell or tissue-engineered medicine.
- PRIME: a priority medicine that the EMA supported early because it may meet an unmet need.
- Conditional approval: approved on less complete data because the need is high; more data must follow.
Key facts
- Brand
- Roctavian
- Active substance
- valoctocogene roxaparvovec
- Medicine group
- Antihemorrhagics
- ATC code
- B02BD15
- Marketing authorisation holder
- BioMarin International Limited
- Authorised in the EU since
- 2022
Official information
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Roctavian used for?
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).
What is the active substance in Roctavian?
Roctavian contains valoctocogene roxaparvovec.
Who makes Roctavian?
The EU marketing authorisation holder is BioMarin International Limited.
Is Roctavian an orphan medicine?
Yes. Roctavian is designated an orphan medicine in the EU, for a rare disease ().
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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