Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Valoctocogene roxaparvovec · 6 trials · 4 indications
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.
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 FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.
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").
Adverse events (AEs) with onset or worsening after the investigational product were included. Participants with more than one AE of the same category were counted only once for that category. Serious adverse event (SAE)
Responder/Non responder status, where a responder was defined as a participant with median FVIII activity of \>= 5 IU/dL during Week 13-16 post-BMN 270 infusion
Values for FVIII activity were excluded from analysis if obtained within 72 hours since the last infusion of exogenous FVIII replacement therapy FVIII activity levels below the Lower limit of quantitation (LLOQ) will be imputed with 0 IU/dL Q1: 25% Percentile; Q3: 75% Percentile
| Arm | Type | Description |
|---|---|---|
| Valoctocogene roxaparvovec | EXPERIMENTAL | Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg |
| Valoctocogene Roxaparvovec Open Label | EXPERIMENTAL | Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg |
| Name | Type | Description |
|---|---|---|
| Valoctocogene roxaparvovec | BIOLOGICAL | Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Severe Hemophilia A |
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...
Valoctocogene roxaparvovec is an investigational gene therapy being developed for the treatment of hemophilia A, including severe hemophilia A and hemophilia A with inhibitors. It is designed to address the underlying genetic cause of the condition by delivering a functional gene to help produce clotting factor VIII.
Valoctocogene roxaparvovec is a gene therapy that works by delivering a copy of the gene for factor VIII, a clotting protein missing or defective in hemophilia A patients. The therapy uses a viral vector to introduce this gene into cells, enabling them to produce functional factor VIII and potentially reduce bleeding episodes.
Valoctocogene roxaparvovec is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. BioMarin is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for hemophilia A.
Valoctocogene roxaparvovec is currently in Phase 3 clinical development. It has completed Phase 1 and Phase 3 trials, with an active Phase 3 study ongoing in Japanese patients. The therapy remains investigational and has not been approved by regulatory authorities.
Valoctocogene roxaparvovec has been studied in four clinical trials, including NCT02576795, a Phase 1 study in severe hemophilia A patients in the UK; NCT03392974, a Phase 3 single-arm study in the US; NCT04684940, a Phase 1 study in hemophilia A with inhibitors; and NCT06224907, an active Phase 3 study in Japanese patients.
Valoctocogene roxaparvovec is also known by the brand name Roctavian, which is the trade name under which it has been studied. No other alternative names have been reported for this gene therapy.