{"id":5,"date":"2023-05-22T11:33:29","date_gmt":"2023-05-22T11:33:29","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/?page_id=5"},"modified":"2025-01-28T11:13:01","modified_gmt":"2025-01-28T11:13:01","slug":"efficacy","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/","title":{"rendered":"Home"},"content":{"rendered":"
\n\t
<\/div>\n\t
<\/div>\n\t
<\/div>\n\n\t
\n\t\t
\n\t\t\t\t\t\t \"It's \t\t<\/div>\n\t\t
\n
\n
\n
\n

PROVEN RESULTS THROUGH 4 YEARS1<\/sup><\/p>\n <\/div>\n

The only one-time gene therapy indicated for the treatment of severe hemophilia A in eligible adults2<\/sup>
\nIndividual results may vary.<\/small><\/p>\n <\/div>\n

\n

Connect with us <\/a><\/p>\n <\/div>\n <\/div>\n <\/div>\n \t\t\t<\/div>\n<\/div>\n\n\n

\n
\n
\n \n
\n

Improved bleed control through 4 years1*<\/sup><\/h2>\n

Mean ABR reduction through 4 years was 37% compared to baseline prophylaxis<\/h3>\n

The imputed mean ABR (SD) was 5.4 (6.9) at baseline vs 3.4 (7.2) after ROCTAVIAN<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

\n\t
\n\t\t
\n\t\t
\n \n \n \n \n
\n
\n \"Mean <\/div>\n <\/figure>\n <\/div>\n \n
\n

What is imputed data?<\/h2>\n

To account for the possibility of no treatment in some patients who returned to prophylaxis, 35 bleeds per year were imputed regardless of the patients’ actual bleed rate.2<\/sup><\/p>\n <\/div>\n \t\t<\/div>\n\t<\/div>\n<\/div>\n\n

\n
\n
\n \n
\n

*Median duration of follow-up was 4.0 years (range: 1.7 to 4.6 years).1<\/sup>
\n\u2020<\/sup>A noninferiority test of the difference in ABR during the EEP following ROCTAVIAN administration was compared with the ABR during the baseline period in the rollover population. The mean difference in ABR was -4.1 (95% CI: -5.4, -2.8) bleeds\/year. The noninferiority analysis met the prespecified margin, indicating the effectiveness of ROCTAVIAN.1,2<\/sup>
\n\u2021<\/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).2<\/sup>
\n\u00a7<\/sup>The 4-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 earliest.2<\/sup>
\n\u2016<\/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.2<\/sup><\/small><\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n
\n \n
\n

Observed bleeds 4 years after ROCTAVIAN (N=112)1*\u2020<\/sup><\/h2>\n <\/div>\n\n
\n
\n
\n\t\t\t \"Observed\t\t\t<\/div>\n\t\t\t
\n\t\t\t \"Observed\t\t\t<\/div>\n <\/div>\n

ABR=annualized bleed rate; EEP, efficacy evaluation period.<\/p>\n

*Median duration of follow-up was 4.0 years (range: 1.7 to 4.6 years).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 at least 6 months on Factor VIII prophylaxis prior to receiving ROCTAVIAN (rollover population). The remaining 22 patients had baseline ABR collected retrospectively (directly enrolled population). All patients were followed for at least 3 years.2<\/sup>
\n\u2021<\/sup>Patients experienced 176 observed spontaneous bleeds during the 6-month baseline evaluation while on Factor VIII prophylaxis; 357 spontaneous bleeds were observed during the EEP.1,2<\/sup>
\n\u00a7<\/sup>Patients experienced 240 observed joint bleeds during the 6-month baseline evaluation while on Factor VIII prophylaxis; 362 joint bleeds were observed during the EEP.1,2<\/sup><\/p>\n<\/figcaption>\n <\/figure>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n
\n \n
\n

Years of sustained Factor VIII levels for the majority of patients1,2*\u2020<\/sup><\/h2>\n

Factor VIII activity level, per OSA<\/h3>\n <\/div>\n\n
\n
\n \"Mean <\/div>\n <\/figure>\n\n
\n

Some patients had increased Factor VIII levels, but it was determined that either they had not responded or had lost response to ROCTAVIAN.<\/p>\n

ABR, annualized bleeding rate; OSA, one-stage assay.<\/small><\/p>\n

*Median duration of follow-up was 4.0 years (range: 1.7 to 4.6 years).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 one-stage assay 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><\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n
\n \n
\n

Patients (%) achieving Factor VIII activity (OSA) thresholds (n=89)1*<\/sup><\/h2>\n <\/div>\n\n
\n
\n
\n\t\t\t \"At\t\t\t<\/div>\n\t\t\t
\n\t\t\t \"At\t\t\t<\/div>\n <\/div>\n <\/figure>\n\n
\n

Ranges reflect World Federation of Hemophilia definitions of hemophilia severity.3<\/sup> Here, we define near-normal to normal as 40 to \u2264150 IU\/dL.<\/p>\n

ABR, annualized bleeding rate; OSA, one-stage assay; ULN, upper limit of normal.<\/small><\/p>\n

*Median duration of follow-up was 4.0 years (range: 1.7 to 4.6 years).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 is not equivalent to response.<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n
\n \n
\n

Most patients eliminated the need for routine prophylaxis1*<\/sup><\/h2>\n <\/div>\n\n
\n
\n \"96% <\/div>\n <\/figure>\n\n
\n

82\/112 responded to ROCTAVIAN and remained off continuous prophylaxis through 4 years.1,2*\u2020\u00a7<\/sup><\/h3>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
\n
\n
\n \n
\n

*Median duration of follow-up was 4.0 years (range: 1.7 to 4.6 years).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>Baseline data were annualized based on a 6-month period of collection.2<\/sup>
\n\u00a7<\/sup>In the rollover population, a total of 5 patients (4%) did not respond and 25 patients (22%) lost response to ROCTAVIAN treatment over a median time of 2.7 (range: 1.0 to 4.1 years). 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 ROCTAVIAN treatment over a median time of 3.8 (range: 1.2 to 4.8 years).1,2
\n<\/sup><\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n
\n \n
\n

References <\/strong>
\n1.<\/strong> Data on file. BioMarin Pharmaceutical Inc.; 2024. 2.<\/strong> ROCTAVIAN\u00ae (valoctocogene roxaparvovec-rvox). Prescribing information. BioMarin International, Ltd.; 2023. 3.<\/strong> Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia<\/em>. 2020;26(Suppl 6):1-158. doi:10.1111\/hae.14046<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n\t\t
\n\t\t\t
\n\t\t\t\t
<\/div>\n\t\t\t\t
\n\t\t\t\t
\n \"\" <\/div>\n \t\t\t\t\t
\n
\n\t\t\t\t\t \t\t\t\t\t \t\t\t\t\t\t

Learn more about the 4-year data for ROCTAVIAN\n<\/h2>\n\t\t\t\t\t\t \t\t\t\t\t\t \t\t\t\t\t\t

Connect with a BioMarin representative.<\/p>\n\t\t\t\t\t\t \t\t\t\t\t <\/div>\n\t\t\t\t\t

\n

Connect with us <\/a><\/p>\n <\/div>\n\t\t\t\t\t <\/div>\n\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-5","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nGene therapy for severe hemophilia A | ROCTAVIAN\u00ae (valoctocogene roxaparvovec-rvox) for Healthcare Professionals<\/title>\n<meta name=\"description\" content=\"ROCTAVIAN\u00ae is the only gene therapy treatment option for adults with severe hemophilia A without active factor VIII inhibitors.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Gene therapy for severe hemophilia A | ROCTAVIAN\u2122 (valoctocogene roxaparvovec-rvox) for Healthcare Professionals\" \/>\n<meta property=\"og:description\" content=\"ROCTAVIAN\u2122 is the only gene therapy treatment option for adults with severe hemophilia A without active factor VIII inhibitors.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/\" \/>\n<meta property=\"og:site_name\" content=\"BioMarin Roctavian HCP EN-US\" \/>\n<meta property=\"article:modified_time\" content=\"2025-01-28T11:13:01+00:00\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:title\" content=\"Gene therapy for severe hemophilia A | ROCTAVIAN\u2122 (valoctocogene roxaparvovec-rvox) for Healthcare Professionals\" \/>\n<meta name=\"twitter:description\" content=\"ROCTAVIAN\u2122 is the only gene therapy treatment option for adults with severe hemophilia A without active factor VIII inhibitors.\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/\",\"url\":\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/\",\"name\":\"Gene therapy for severe hemophilia A | ROCTAVIAN\u00ae (valoctocogene roxaparvovec-rvox) for Healthcare Professionals\",\"isPartOf\":{\"@id\":\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/#website\"},\"datePublished\":\"2023-05-22T11:33:29+00:00\",\"dateModified\":\"2025-01-28T11:13:01+00:00\",\"description\":\"ROCTAVIAN\u00ae is the only gene therapy treatment option for adults with severe hemophilia A without active factor VIII inhibitors.\",\"breadcrumb\":{\"@id\":\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/\"]}]},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Home\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/#website\",\"url\":\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/\",\"name\":\"BioMarin Roctavian HCP EN-US\",\"description\":\"\",\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/?s={search_term_string}\"},\"query-input\":\"required name=search_term_string\"}],\"inLanguage\":\"en-US\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"Gene therapy for severe hemophilia A | ROCTAVIAN\u00ae (valoctocogene roxaparvovec-rvox) for Healthcare Professionals","description":"ROCTAVIAN\u00ae is the only gene therapy treatment option for adults with severe hemophilia A without active factor VIII inhibitors.","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/","og_locale":"en_US","og_type":"article","og_title":"Gene therapy for severe hemophilia A | ROCTAVIAN\u2122 (valoctocogene roxaparvovec-rvox) for Healthcare Professionals","og_description":"ROCTAVIAN\u2122 is the only gene therapy treatment option for adults with severe hemophilia A without active factor VIII inhibitors.","og_url":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/","og_site_name":"BioMarin Roctavian HCP EN-US","article_modified_time":"2025-01-28T11:13:01+00:00","twitter_card":"summary_large_image","twitter_title":"Gene therapy for severe hemophilia A | ROCTAVIAN\u2122 (valoctocogene roxaparvovec-rvox) for Healthcare Professionals","twitter_description":"ROCTAVIAN\u2122 is the only gene therapy treatment option for adults with severe hemophilia A without active factor VIII inhibitors.","schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"WebPage","@id":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/","url":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/","name":"Gene therapy for severe hemophilia A | ROCTAVIAN\u00ae (valoctocogene roxaparvovec-rvox) for Healthcare Professionals","isPartOf":{"@id":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/#website"},"datePublished":"2023-05-22T11:33:29+00:00","dateModified":"2025-01-28T11:13:01+00:00","description":"ROCTAVIAN\u00ae is the only gene therapy treatment option for adults with severe hemophilia A without active factor VIII inhibitors.","breadcrumb":{"@id":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/hcp.biomarin.com\/en-us\/roctavian\/"]}]},{"@type":"BreadcrumbList","@id":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/"},{"@type":"ListItem","position":2,"name":"Home"}]},{"@type":"WebSite","@id":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/#website","url":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/","name":"BioMarin Roctavian HCP EN-US","description":"","potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/?s={search_term_string}"},"query-input":"required name=search_term_string"}],"inLanguage":"en-US"}]}},"_links":{"self":[{"href":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/wp-json\/wp\/v2\/pages\/5","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/wp-json\/wp\/v2\/comments?post=5"}],"version-history":[{"count":182,"href":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/wp-json\/wp\/v2\/pages\/5\/revisions"}],"predecessor-version":[{"id":3237,"href":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/wp-json\/wp\/v2\/pages\/5\/revisions\/3237"}],"wp:attachment":[{"href":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/wp-json\/wp\/v2\/media?parent=5"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}