{"id":16,"date":"2023-05-22T12:06:40","date_gmt":"2023-05-22T12:06:40","guid":{"rendered":"https:\/\/roctavianhcp-dev-001.azurewebsites.net\/en-us-new\/roctavian\/?page_id=16"},"modified":"2023-12-21T09:52:19","modified_gmt":"2023-12-21T09:52:19","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\n<\/h1>\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n
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One-time ROCTAVIAN. Improved bleed control1<\/sup><\/h2>\n

Proven reduction in ABR from baseline prophylaxis*1<\/sup><\/h3>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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SD=standard deviation.
\n*An NI test of the difference in ABR during the EEP following ROCTAVIAN administration was compared with ABR during the baseline period in the rollover population. The mean difference in ABR was -2.8 (95% CI: -4.3, -1.2) bleeds\/year. The NI analysis met the prespecified margin, indicating the effectiveness of ROCTAVIAN.
\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 population).
\n\u2021<\/sup>A total of 13 patients (12%) had used Factor VIII replacement products or emicizumab during the efficacy evaluation period for prophylaxis, with a median start time at 2.3 (range: 0.1 to 3.3) years. An ABR of 35 was imputed for the periods when these patients were on prophylaxis.
\n\u00a7<\/sup>The 3-year follow-up period started from Study Day 33 (Week 5) or the end of Factor VIII prophylaxis including a washout period after ROCTAVIAN treatment, 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 the earliest.
\n\u00b6<\/sup>Median duration of follow-up was 3.0 years (range: 1.7, 3.7).<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Study design\n<\/h2>\n <\/div>\n
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Phase 3 study design<\/strong><\/p>\n