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Valoctocogene Roxaparvovec

Hemophilia A

Explore 62 assets in Hemophilia A →
Floatz Rating
B62/100
Confidence
Indicative
v0.2
Sponsor
BioMarin Pharmaceutical
Modality
gene_therapy
Development Phase
Phase 3
Status
Active
Evidence ledger · v0.2

Clinical Evidence

Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
59High confidence
Detailed axis rationale is planned and will be published soon.
TrialPhaseStatusNPrimary endpointReadout
NCT06224907CT.gov
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
—Active Not Recruiting———
NCT04684940CT.gov
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
—Active Not Recruiting———
NCT04323098CT.gov
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
—Completed———
NCT03520712CT.gov
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
—Terminated———
NCT03392974CT.gov
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
—Completed———
NCT03370913CT.gov
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
—Completed———

Competitive Position

Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
73High confidence
Detailed axis rationale is planned and will be published soon.

Same indication · Hemophilia A

AssetSponsorPhaseRating
Valoctocogene Roxaparvovec (this asset)BioMarin PharmaceuticalP3B · 62
Turoctocog AlfaNovo Nordisk A/SP4A
Eptacog Alfa (Activated)AryoGen Pharmed Co.P4A
Rurioctocog Alfa PegolTakedaP4BBB
bay94-9027BayerP4BBB
Immunoglobulin G4 [230-Proline,De-449-Lysine], Anti-(Human Blood-Coagulation Factor Ix)(Human Clone 0365-0001-9985 ?4-Chain), Disulfide With Human Clone 0365-0001-9985 ?-Chain, (228?226?),(231?229?)-Bis(Disulfide) With Anti-(Human Blood-Coagulation Factor X) Immunoglobulin G4 [228-Proline,405-Leucine,409-Lysine,De-447-Lysine] (Human Clone 0365-0001-8174 ?4-Chain) Disulfide With Human Clone 0365-0001-8174 ?-ChainNovo Nordisk A/SP3BBB
N8-GPNovo Nordisk A/SP3BBB
EmicizumabEmory UniversityP4BBB
FitusiranSanofiP4BBB

+36 more in the Hemophilia A cohort

Other indications for Valoctocogene Roxaparvovec

IndicationSponsorPhaseRating
Severe Hemophilia A—P1/2CCC · 52
Blood Coagulation Disease—P1/2CCC · 48
Hematologic Disorder—P1/2CCC · 48

Scientific Foundation

Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR

Planned for methodology v0.2.

Development Feasibility

How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR

Planned for methodology v0.2.

Commercial Opportunity

Commercial prize: addressable population, unmet need, and the value case for the indication.
NR

Planned for methodology v0.2.

IP & Exclusivity

Exclusivity position, covering patent protection and freedom-to-operate runway.
NR

Planned for methodology v0.2.

Manufacturing & Supply

Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR

Planned for methodology v0.2.

Related assets

Citation

Floatz Terminal. Valoctocogene Roxaparvovec in Hemophilia A. Methodology v0.2.
Rated under v0.2 effective July 8, 2026. Last refreshed July 11, 2026.
Accessed July 14, 2026.
https://terminal.floatz.ai/assets/valoctocogene-roxaparvovec-hemophilia-a

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