Friday, July 10, 2026

Drug profile · INN

valoctocogene roxaparvovec

valoctocogene roxaparvovec (ROCTAVIAN) is a coagulation factor ix exogenous protein. Associated with BioMarin Pharmaceutical Australia Pty Ltd.

EMA: approved Blood and blood forming organs (B02) B02BD15
Patents linked 0
Pipeline programs 4
News articles 1
Drug details — valoctocogene roxaparvovec

Quick answer

valoctocogene roxaparvovec (ROCTAVIAN) is a coagulation factor ix exogenous protein. Associated with BioMarin Pharmaceutical Australia Pty Ltd.

Key facts

INN valoctocogene roxaparvovec
Brand ROCTAVIAN
Mechanism Coagulation factor IX exogenous protein
Target Coagulation factor IX
Therapeutic class Blood and blood forming organs (B02)
ATC code B02BD15

Mechanism of action

Coagulation factor IX exogenous protein

Primary target: Coagulation factor IX

Approval history

Authority Status Date
EMA approved

Recent coverage

Pipeline programs

  1. valoctocogene roxaparvovec Phase 3 · BioMarin Pharmaceutical Australia · Hemophilia A
  2. Valoctocogene roxaparvovec Phase 2 · BioMarin Pharmaceutical Australia · Hemophilia A With Inhibitor
  3. valoctocogene roxaparvovec Phase 2 · BioMarin Pharmaceutical Australia · Severe Haemophilia A
  4. ROCTAVIAN 2 × 1013 vector genomes/mL solution for infusion Approved · BioMarin Pharmaceutical Australia · Hemophilia A

Related & competitor drugs

  1. apixaban Blood and blood forming organs (B01)
  2. berotralstat Blood and blood forming organs (B06)
  3. bivalirudin Blood and blood forming organs (B01)
  4. c1 inhibitor (human) Blood and blood forming organs (B06)
  5. clopidogrel Blood and blood forming organs (B01)
  6. dabigatran etexilate Blood and blood forming organs (B01)

Frequently asked questions

What is valoctocogene roxaparvovec?

valoctocogene roxaparvovec (ROCTAVIAN) is a coagulation factor ix exogenous protein. Associated with BioMarin Pharmaceutical Australia Pty Ltd.

What is the brand name for valoctocogene roxaparvovec?

ROCTAVIAN is a marketed brand name for valoctocogene roxaparvovec. Check FDA Drugs@FDA for the current US label holder and prescribing information.