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Valoctocogene roxaparvovec

Phase 3

Hemophilia A | Monoclonal antibody | Hematology |BioMarin Pharmaceutical Inc.|Last Updated: Mar 19, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials4
Total Enrollment173
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06224907Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia APHASE3 ACTIVE NOT_RECRUITING 6Dec 25, 2023Mar 1, 2029Mar 19, 20264 Japan
NCT04323098Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia APHASE3 COMPLETED 22Dec 8, 2020May 8, 2025Oct 8, 202512 United States, Australia +2
NCT03392974Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kgPHASE3 COMPLETED 1Mar 14, 2018Jun 5, 2023Oct 3, 20231 United States
NCT03370913Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)PHASE3 COMPLETED 144Dec 19, 2017Nov 20, 2024Mar 25, 202549 United States, Australia +11
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Study Endpoints
Primary Endpoints
Change in the human coagulation factor VIII (hFVIII) activity, as measured by chromogenic substrate assay, during Weeks 49 to 52 post BMN 270 infusion from Baseline.
52 Weeks

The change from baseline in FVIII activity, as measured by chromogenic substrate assay, during Weeks 49 to 52 post-BMN 270 infusion. Each subject's FVIII activity level during Week 49 to 52 is defined as the median of the values obtained during week 49-52 with the analysis window defined. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis. The baseline value is imputed as 1 IU/dL for each subject.

Change From Baseline in FVIII Activity as Measured by Chromogenic Substrate Assay at Week 52.
Baseline to Week 52

The change from baseline (assuming no treatment for severe hemophilia A) in FVIII activity, as measured by chromogenic substrate assay (CSA), at Week 52 (during Weeks 49 - 52) post-BMN 270 infusion. Each participant's FVIII activity level at Week 52 is defined as the median of the values obtained within the analysis window at Weeks 49-52. The baseline value will be imputed as 1 IU/dL, since there will be no washout of severe hemophilia A participants' usual FVIII prophylaxis (in order to avoid increasing the risk of bleeding) prior to BMN 270 infusion. Post-BMN 270 infusion values for FVIII activity will be excluded from analysis if obtained within 72 hours (or 3 calendar days if time is not available) since the last infusion of exogenous FVIII replacement therapy. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis.

Change of the Median Factor VIII (FVIII) Activity
Week 52

Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.

Change From Baseline in Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment [Annualized Bleeding Rate (ABR) for All Bleeds] in EEP.
Baseline to efficacy evaluation period (EEP)

All bleeds comprises both treated and non-treated bleeds. In this definition, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. All bleeds are any reported bleeding events regardless of the use of FVIII or other treatments. ABR for all bleeds= Number of bleeding episodes for all bleeds during the calculation period / total number of days during the calculation period \* 365.25. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis. EEP: From Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for the 2-year analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit").

Secondary Endpoints
Change in the annualized utilization (IU/kg/year) of exogenous FVIII replacement therapy in the efficacy evaluation period (EEP) ("Post FVIII Prophylaxis to Last Visit") from the Baseline utilization of exogenous FVIII replacement therapy.
Baseline to at least Week 52
Change in the annualized bleeding rate (i.e., number of bleeding episodes per year) requiring exogenous FVIII replacement treatment therapy in the efficacy evaluation period ("Post FVIII Prophylaxis to Last Visit") from Baseline.
Baseline to at least Week 52
Change from Baseline in Hemophilia-Specific Health-Related Quality of Life Questionnaire for Adults (Haemo-QoL-A) total score, physical functioning, role functioning, and consequences of bleeding domain scores at Week 52 of study post-BMN 270 infusion
Baseline to Week 52
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Valoctocogene roxaparvovecEXPERIMENTALSingle administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg
Valoctocogene Roxaparvovec Open LabelEXPERIMENTALSingle administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg
Interventions
NameTypeDescription
Valoctocogene roxaparvovecBIOLOGICALAdeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Severe Hemophilia A
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Eligibility Criteria
Age Range18 Years — N/A
SexMALE
Healthy VolunteersYes
Study Sites4

Inclusion Criteria: * Japanese males ≥18 years of age with HA and endogenous FVIII activity levels \<1 IU/dL as evidenced by medical history, at the time of signing the informed consent * Must have been on prophylactic FVIII replacement therapy for at least 12 months prior to study entry. High-qual...

Countries:JapanUnited StatesAustraliaBrazilTaiwanBelgiumFranceGermanyIsraelItalySouth AfricaSouth KoreaSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06224907primaryCompletionDate: changed
LOWMay 24, 2026NCT06224907studyFirstPostDate: changed