{"id":18,"date":"2023-05-22T12:07:02","date_gmt":"2023-05-22T12:07:02","guid":{"rendered":"https:\/\/roctavianhcp-dev-001.azurewebsites.net\/en-us-new\/roctavian\/?page_id=18"},"modified":"2023-12-21T09:53:57","modified_gmt":"2023-12-21T09:53:57","slug":"safety","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/safety\/","title":{"rendered":"Safety"},"content":{"rendered":"
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SAFETY PROFILE\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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The safety of ROCTAVIAN was evaluated in 134 patients in the Phase 3 study (112 rollover and 22 directly enrolled). Long-term safety continues to be evaluated in the ongoing clinical trial. Follow-up is scheduled to last through 5 years. Below, please see the most common adverse reactions for patients treated with ROCTAVIAN.<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Adverse reactions in \u22655%a<\/sup> of patients treated with ROCTAVIAN<\/strong><\/h4>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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Adverse Reactions<\/th>Number of Patients (%)
N=134<\/th><\/tr>
<\/th>All Grades<\/th>\u2265Grade 3<\/th><\/tr><\/thead>
Nervous system disorders<\/th><\/tr>
Headache<\/td>9 (7%)<\/td>0 (0%)<\/td><\/tr>
Gastrointestinal disorders<\/th><\/tr>
Nausea<\/td>42 (31%)<\/td>0 (0%)<\/td><\/tr>
Vomiting<\/td>8 (6%)<\/td>0 (0%)<\/td><\/tr>
Abdominal painb<\/sup><\/small><\/td>8 (6%)<\/td>0 (0%)<\/td><\/tr>
General disorders and administration site conditions<\/th><\/tr>
Fatiguec<\/sup><\/small><\/td>21 (16%)<\/td>0 (0%)<\/td><\/tr>
Infusion-related reactionsd<\/sup><\/small><\/td>9 (7%)<\/td>2 (1%)<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n
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a<\/sup>Other adverse reactions include gastroenteritis (2 patients; 1%), rash (2 patients; 1%), diarrhea (6 patients; 4%), and dizziness (3 patients; 2%).
\nb<\/sup>Includes abdominal discomfort, abdominal distension, abdominal tenderness, and abdominal pain upper.
\nc<\/sup>Includes fatigue, lethargy, and malaise.
\nd<\/sup>Infusion-related reactions are not under a specific system organ class and include multiple symptoms that occurred during or within 6 hours after the end of infusion.<\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Contraindications<\/h3>\n

Patients with active infections, either acute (such as acute respiratory infections or acute hepatitis) or uncontrolled chronic (such as chronic active hepatitis B). Patients with known significant hepatic fibrosis (stage 3 or 4 on the Batts-Ludwig scale or equivalent), or cirrhosis, and patients with known hypersensitivity to mannitol.<\/p>\n

Warnings and Precautions<\/h3>\n

Infusion-related reactions<\/strong> including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during and for at least 3 hours after ROCTAVIAN administration. Administer ROCTAVIAN in a setting where personnel and equipment are immediately available to treat infusion-related reactions. Discontinue infusion for anaphylaxis.<\/p>\n

Hepatotoxicity:<\/strong> The safety and effectiveness of ROCTAVIAN in patients with hepatic impairment has not been established. Perform liver health assessments prior to administration. The majority of patients treated with ROCTAVIAN experienced ALT elevations and required corticosteroids for ALT elevation. Assess patient\u2019s ability to receive corticosteroids and\/or other immunosuppressive therapy that may be required for an extended period. Live vaccines should not be administered to patients while on immunosuppressive therapy.<\/p>\n

Monitor ALT weekly for at least 26 weeks and as clinically indicated, during corticosteroid therapy and institute corticosteroid treatment in response to ALT elevations as required. Continue to monitor ALT until it returns to baseline. Monitor Factor VIII activity levels since ALT elevation may be accompanied by a decrease in Factor VIII activity. One case of autoimmune hepatitis was reported during third year follow-up in a patient with history of hepatitis C and steatohepatitis.<\/p>\n

It is recommended that patients abstain from consuming alcohol for at least 1 year after administration and thereafter limit alcohol use. Concomitant medications may cause hepatotoxicity, decrease Factor VIII activity, or change plasma corticosteroid levels which may impact liver enzyme elevation and\/or Factor VIII activity or decrease the efficacy of the corticosteroid regimen or increase their side effects. Closely monitor concomitant medication use including herbal products and nutritional supplements and consider alternative medications in case of potential drug interactions.<\/p>\n

Thromboembolic Events:<\/strong> Factor VIII activity above ULN has been reported following ROCTAVIAN infusion. Thromboembolic events may occur in the setting of elevated Factor VIII activity above ULN. Evaluate patients for risk of thrombosis including general cardiovascular risk factors before and after administration of ROCTAVIAN. Advise patients on their individual risk of thrombosis in relation to their Factor VIII activity levels above ULN and consider prophylactic anticoagulation. Advise patients to seek immediate medical attention for signs or symptoms indicative of a thrombotic event.<\/p>\n

Factor VIII Inhibitors and Monitoring for Inhibitors.<\/strong> The safety and effectiveness of ROCTAVIAN in patients with prior or active Factor VIII inhibitors have not been established. Patients with active Factor VIII inhibitors should not take ROCTAVIAN. Following administration, monitor patients for Factor VIII inhibitors (neutralizing antibodies to Factor VIII). Test for Factor VIII inhibitors especially if bleeding is not controlled, or plasma Factor VIII activity levels decrease.<\/p>\n

Monitor Factor VIII<\/strong> using the same schedule for ALT monitoring. It may take several weeks after ROCTAVIAN infusion before ROCTAVIAN-derived Factor VIII activity rises to a level sufficient for prevention of spontaneous bleeding episodes. Exogenous Factor VIII or other hemostatic products may also be required in case of surgery, invasive procedures, trauma, or bleeds. Consider more frequent monitoring in patients with Factor VIII activity levels \u22645 IU\/dL and evidence of bleeding, taking into account the stability of Factor VIII levels since the previous measurement.<\/p>\n

Factor VIII activity produced by ROCTAVIAN in human plasma is higher if measured with one-stage clotting assays compared to chromogenic substrate assays. When switching from hemostatic products prior to ROCTAVIAN treatment, physicians should refer to the relevant prescribing information to avoid the potential for Factor VIII activity assay interference during the transition period.<\/p>\n

Malignancy:<\/strong> The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. ROCTAVIAN can also insert into the DNA of other human body cells. Monitor patients with risk factors for hepatocellular carcinoma (eg, hepatitis B or C, nonalcoholic fatty liver disease, chronic alcohol consumption, nonalcoholic steatohepatitis, advanced age) with regular liver ultrasound (eg, annually) and alpha-fetoprotein testing for 5 years following ROCTAVIAN administration. In the event that any malignancy occurs after treatment with ROCTAVIAN, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100.<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Laboratory abnormalities1<\/sup><\/h2>\n <\/div>\n\n
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Laboratory abnormalities (\u226510%) in patients treated with ROCTAVIAN (N=134)<\/strong><\/h4>\n <\/div>\n\n\n
ALT Increases >ULN<\/strong><\/td>109 (81%)<\/td><\/tr>
AST Increases >ULN<\/strong><\/td>92 (69%)<\/td><\/tr>
LDH Increases >ULN<\/strong><\/td>77 (57%)<\/td><\/tr>
CPK Increases<\/strong> >ULN<\/strong><\/td>60 (45%)<\/td><\/tr>
Factor VIII Activity Levels >ULN<\/strong>a<\/sup><\/td>38 (28%)<\/td><\/tr>
GGT Increases >ULN <\/strong><\/td>24 (18%)<\/td><\/tr>
Bilirubin Increases >ULN <\/strong><\/td>18 (13%)<\/td><\/tr><\/tbody><\/table>
ALT=alanine aminotransferase; AST=aspartate aminotransferase; CPK=creatine phosphokinase; GGT=gamma-glutamyl transferase; LDH=lactate dehydrogenase.
<\/small>a<\/sup>Patients with 1 or more instances of Factor VIII activity levels >170 IU\/dL (ULN of the chromogenic assay used) or >150 IU\/dL (ULN of the one-stage assay used).<\/figcaption><\/figure>\n\n <\/div>\n <\/div>\n<\/div>\n\n
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Steps to treatment\n<\/h2>\n\t\t\t\t\t\t \t\t\t\t\t\t \t\t\t\t\t\t

Find out the requirements for ROCTAVIAN<\/p>\n\t\t\t\t\t\t \t\t\t\t\t <\/div>\n\t\t\t\t\t

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view criteria <\/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>\n\n

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Reference:\n<\/h4>\n\t\t\t\t\t\t
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  1. ROCTAVIAN\u2122 (valoctocogene roxaparvovec-rvox). Prescribing Information. BioMarin Pharmaceutical Inc.; 2023.\n<\/span><\/li>\n <\/ol>\n\t\t\t\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":""},"acf":[],"yoast_head":"\nSafety | ROCTAVIAN\u2122 (valoctocogene roxaparvovec-rvox) for Healthcare Professionals<\/title>\n<meta name=\"description\" content=\"Review safety data for ROCTAVIAN\u2122 (valoctocogene roxaparvovec-rvox) which includes important information on tolerability, hepatic reactions, and adverse reactions\" \/>\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\/safety\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" 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