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As of May 29th, 2026, BioMarin has voluntarily withdrawn ROCTAVIAN (valoctocogene roxaparvovec-rvox) from the market

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Real World Evidence

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Publications
Hemophilia A
2025
Real World Evidence

Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance

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Safety Profile and Efficacy

Plain Language Summaries

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Real World Evidence

Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance

Safety Profile and Efficacy

Plain language summary of 4-year results of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia A

Comparative effectiveness of valoctocogene roxaparvovec and efanesoctocog alfa in the treatment of severe hemophilia A: A matching-adjusted indirect comparison of bleeding frequency

Estimated long-term durability of valoctocogene roxaparvovec treatment in male patients with severe hemophilia A: An extrapolation of clinical data

Safety and efficacy of valoctocogene roxaparvovec in participants with active or prior FVIII inhibitors: Results from a phase 1/2 trial

Bleeding rate, FVIII utilization, corticosteroid use, quality of life and safety 4 years after gene transfer with valoctocogene roxaparvovec in a Brazilian cohort: Results from GENEr8-1

Health-related quality of life improvements following valoctocogene roxaparvovec gene therapy in people without bleeds or target joints at baseline: a post hoc analysis from GENEr8-1

A plain language summary on indirectly comparing bleeding after valoctocogene roxaparvovec gene therapy to bleeding with emicizumab prophylaxis

Bleeding Outcomes in Participants With Factor VIII Activity <5 IU/dL Post-Gene Transfer in GENEr8-1

Hemostatic results for up to 6 years following treatment with valoctocogene roxaparvovec, an AAV5-hFVIII-SQ gene therapy for severe hemophilia A

Patient Reported Outcomes

Bleeding rate, FVIII utilization, corticosteroid use, quality of life and safety 4 years after gene transfer with valoctocogene roxaparvovec in a Brazilian cohort: Results from GENEr8-1

Health-related quality of life improvements following valoctocogene roxaparvovec gene therapy in people without bleeds or target joints at baseline: a post hoc analysis from GENEr8-1

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