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Roctavian

Approved

Valoctocogene Roxaparvovec

BioMarin Pharmaceutical Inc. (BMRN)
Orphan DrugFast TrackBreakthrough TherapyLearn about designations

Approved

Development Phase

0

Upcoming Catalysts

0

Historical Events

2

Regulatory Approvals

No Catalysts Found

No catalyst events have been recorded for this drug yet.

Competitive Landscape
Drug Information

Target Indication

Hemophilia A

Mechanism of Action

Gene Therapy

Clinical Trial

NCT03370913

Last updated: 12/3/2025

Patent & Exclusivity

No patent data available. Pre-approval drugs may not have Orange Book listings.

Regulatory Approvals
EMAPending

Roctavian

Treatment of haemophilia A

Orphan
View on EMA
EMA8/24/2022

Roctavian

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).

OrphanConditionalPRIME
View on EMA
Drug Science
Mechanism of action & safety profile
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