{"id":3273,"date":"2025-10-23T13:24:47","date_gmt":"2025-10-23T13:24:47","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/?page_id=3273"},"modified":"2025-12-15T13:10:27","modified_gmt":"2025-12-15T13:10:27","slug":"efficacy","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/efficacy\/","title":{"rendered":"Efficacy"},"content":{"rendered":"
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EFFICACY DATA FOR ROCTAVIAN\n<\/h1>\n <\/div>\t\t\n
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Discover why patients have chosen ROCTAVIAN<\/p>\n <\/div>\n

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Watch their stories<\/a><\/p>\n <\/div>\n <\/div>\n <\/div>\n

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Andrew S., dosed with ROCTAVIAN in 2018 as part of a clinical trial.<\/p>\n <\/div>\n\n

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In the clinical trial, most patients achieved years of endogenous Factor VIII production1,2<\/span><\/sup><\/h2>\n

5 years after ROCTAVIAN, the mean (SD) Factor VIII activity level (per OSA) was 29.8% (48.6)*\u2020<\/sup><\/h4>\n <\/div>\n\n
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OSA, one-stage assay.
\n*<\/sup>Median duration of follow-up was 5.0 years (range: 1.7 to 5.2).1<\/sup>
\n\u2020<\/sup>Of the 134 patients who received ROCTAVIAN in the clinical trial, 112 patients had baseline ABR data prospectively collected during a period of \u22656 months on Factor VIII prophylaxis prior to receiving ROCTAVIAN (rollover population). The remaining 22 patients had baseline ABR collected retrospectively (directly enrolled).2<\/sup>
\n\u2021<\/sup>Factor VIII activity produced by ROCTAVIAN in human plasma is higher if measured with OSA compared to chromogenic assay. In clinical trials, there was a high correlation between one-stage and chromogenic Factor VIII activity levels across the entire range of each assay\u2019s results. For routine clinical monitoring of Factor VIII activity levels, either assay may be used. The conversion factor between the assays can be approximated based on clinical trial results (central laboratory) to be: OSA=1.5 X CSA.2<\/sup><\/p>\n<\/figcaption>\n <\/figure>\n <\/div>\n <\/div>\n<\/div>\n\n

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5 years after ROCTAVIAN, 84% of patients with severe hemophilia A at baseline had Factor VIII activity levels \u22655% (n=85)*\u2020\u2021\u00a7<\/sup><\/h2>\n

Most patients had Factor VIII (OSA) levels in the mild to normal range (n=85)\u2021\u00a7<\/span><\/sup><\/h4>\n <\/div>\n\n
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*<\/sup>Median duration of follow-up was 5.0 years (range: 1.7, 5.2).1<\/sup>
\n\u2020<\/sup>Of the 134 patients who received ROCTAVIAN in the clinical trial, 112 patients had baseline ABR data prospectively collected during a period of \u22656 months on Factor VIII prophylaxis prior to receiving ROCTAVIAN (rollover population). The remaining 22 patients had baseline ABR collected retrospectively (directly enrolled).2<\/sup>
\n\u2021<\/sup>Available data from rollover population (n=112) at Week 260.
\n\u00a7<\/sup>Factor VIII activity is not equivalent to response.
\n||<\/sup>Patients in clinical trial experienced Factor VIII levels of <1%. Under the 5-year analysis of available, valid data, the range was 1.5 to 321.2.<\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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\u201cI would describe my experience with ROCTAVIAN as eye-opening. It\u2019s pretty cool to know that my body is making its own factor now.\u201d<\/p>\n

\u2014Andrew S., dosed with ROCTAVIAN in 2018 as part of a clinical trial<\/cite>\n<\/p><\/blockquote>\n <\/div>\n \n

\n Play<\/a>\n <\/div>\n \n

Watch his story<\/a><\/p> \n <\/div>\n\t<\/div>\n<\/div>\n\n

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In the clinical trial, ROCTAVIAN improved bleed control from baseline for 5 years with a single infusion1,2*\u2020\u2021\u00a7\u2225\u00b6<\/sup><\/h2>\n

The imputed mean ABR (SD) was 5.4 (6.9) at baseline vs 3.9 (7.9) after ROCTAVIAN<\/p>\n <\/div>\n\n

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*<\/sup>The imputed mean ABR (SD) was 5.4 (6.9) at baseline vs 3.9 (7.9), and the imputed median ABR was 3.3 (range: 0, 34.6) at baseline vs 0.4 (range: 0, 35) after ROCTAVIAN.2<\/sup>
\n\u2020<\/sup>Median duration of follow-up was 5.0 years (range: 1.7, 5.2).1<\/sup>
\n\u2021<\/sup>A noninferiority (NI) test of the difference in ABR during the EEP following administration of ROCTAVIAN was compared with the ABR during the baseline period in the rollover population. The mean difference in ABR was -1.44 (95% CI: -3.28, 0,40) bleeds\/year. The NI analysis met the prespecified margin, indicating the effectiveness of ROCTAVIAN.1,2<\/sup>
\n\u00a7<\/sup>Of the 134 patients who received ROCTAVIAN in the clinical trial, 112 patients had baseline ABR data prospectively collected during a period of \u22656 months on Factor VIII prophylaxis prior to receiving ROCTAVIAN (rollover population). The remaining 22 patients had baseline ABR collected retrospectively (directly enrolled).1<\/sup>
\n\u2016<\/sup>The 5-year follow-up period started from Study Day 33 (Week 5) or at the end of Factor VIII prophylaxis, including a washout period after treatment with ROCTAVIAN, whichever was later, and ended when a patient completed the study, had the last visit, or withdrew or was lost to follow-up from the study, whichever was earliest.1<\/sup>
\n\u00b6<\/sup>There were 19 patients (17%) from the 112 rollover population who returned to prophylaxis with median start time at 984 days (range: 33, 1467). An ABR of 35 was imputed for the periods when these patients were on prophylaxis.1<\/sup><\/p>\n<\/figcaption>\n <\/figure>\n <\/div>\n <\/div>\n<\/div>\n\n

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Most patients experienced a >75% reduction across multiple bleed types 5 years after ROCTAVIAN1<\/sup><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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ROCTAVIAN reduced annualized infusions by 95% through 5 years1,2*<\/sup><\/h2>\n <\/div>\n\n
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71% (80\/112) of patients in the rollover population responded to ROCTAVIAN and remained off continuous prophylaxis through 5 years<\/h4>\n <\/div>\n\n
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*<\/sup>Median duration of follow-up was 5.0 years (range: 1.7, 5.2).1<\/sup>
\n<\/small>\u2020<\/sup>Of the 134 patients who received ROCTAVIAN in the clinical trial, 112 patients had baseline ABR data prospectively collected during a period of \u22656 months on Factor VIII prophylaxis prior to receiving ROCTAVIAN (rollover population). The remaining 22 patients had baseline ABR collected retrospectively (directly enrolled).
\n<\/small>\u00a7<\/sup>Baseline data were annualized based on a 6-month period of collection.2<\/sup>
\n<\/small>\u2021<\/sup>In the rollover population, a total of 5 patients (4%) did not respond and 27 patients (24%) lost response to treatment with ROCTAVIAN over a median time of 2.7 years (range: 0.96, 4.54). In the directly enrolled population with a longer follow-up, a total of 1 patient (5%) did not respond and 8 patients (36%) lost response to treatment with ROCTAVIAN over a median time of 3.8 years (range: 1.2, 4.8). In the clinical trial, patients who did not respond or lost response returned to prophylaxis.1<\/sup><\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Health-related quality of life changes were measured over 5 years1,4<\/sup><\/h2>\n

The Haemo-QoL-A (Haemophilia-Specific Quality of Life Questionnaire) is designed to measure health-related quality of life specifically for people with hemophilia<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Haemo-QoL-A measures included:<\/p>\n