{"id":18,"date":"2023-05-22T12:07:02","date_gmt":"2023-05-22T12:07:02","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/?page_id=18"},"modified":"2025-01-28T11:12:33","modified_gmt":"2025-01-28T11:12:33","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 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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Safety has been established in clinical trials<\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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Contraindictions<\/h2>\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

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Select Warnings and Precautions<\/h2>\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> Monitor ALT. 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.<\/p>\n

Monitor for Factor VIII<\/strong> activity and Factor VIII inhibitors.<\/p>\n

Malignancy:<\/strong> Monitor for hepatocellular malignancy in patients with risk factors for hepatocellular carcinoma (eg, hepatitis B or C, nonalcoholic fatty liver disease, chronic alcohol consumption, nonalcoholic steatohepatitis, advanced age). Perform regular liver ultrasound (eg, annually) andalpha-fetoprotein testing following administration.<\/p>\n

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 events for patients treated with ROCTAVIAN.<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Adverse reactions in \u22655% of patients treated with ROCTAVIAN1*<\/sup><\/h3>\n <\/div>\n\n\n



Adverse Reactions<\/th>		Number of Patients (%)
N=134<\/th><\/tr>
All Grades<\/th>\u2265Grade 3<\/th><\/tr><\/thead>
Nervous system disorders<\/th><\/tr>
Headache<\/td>10 (7.5%)<\/td>0 (0%)<\/td><\/tr>
Gastrointestinal disorders<\/th><\/tr>
Nausea<\/td>43 (32%)<\/td>0 (0%)<\/td><\/tr>
Vomiting<\/td>8 (6%)<\/td>0 (0%)<\/td><\/tr>
Abdominal pain\u2020<\/sup><\/small><\/sup><\/small><\/td>8 (6%)<\/td>0 (0%)<\/td><\/tr>
General disorders and administration site conditions<\/th><\/tr>
Fatigue\u2021<\/sup><\/small><\/td>21 (16%)<\/td>0 (0%)<\/td><\/tr>
Infusion-related reactions\u00a7<\/sup><\/small><\/sup><\/small><\/td>2 (2%)<\/td>0 (0%)<\/td><\/tr><\/tbody><\/table>
ABR=annualized bleed rate; EEP, efficacy evaluation period. <\/small>

*Other adverse reactions include gastroenteritis (1 patient; 1%), rash (4 patients; 3%), diarrhea (6 patients; 5%), and dizziness (3 patients; 2%).1,2<\/sup> <\/small>
\u2020<\/sup>Includes abdominal discomfort, abdominal distension, abdominal tenderness, and abdominal pain upper.2<\/sup> <\/small>
\u2021<\/sup>Includes fatigue, lethargy, and malaise.2<\/sup> <\/small>
\u00a7<\/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.2<\/sup><\/small><\/figcaption><\/figure>\n\n <\/div>\n <\/div>\n<\/div>\n\n
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Most ALT elevations occurred in the first 26 weeks post-infusion and were asymptomatic.<\/h2>\n

ALT elevations were resolved with temporary corticosteroids. Treatment with corticosteroids in response to ALT elevations were generally transient with a median duration (range) of 35 weeks (3, 120 weeks).2<\/sup><\/p>\n

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Laboratory abnormalities (10%) in patients treated with ROCTAVIAN (N=134)1<\/sup><\/strong><\/h3>\n <\/div>\n\n\n
ALT Increases >ULN<\/strong><\/td>110 (82%)<\/td><\/tr>
AST Increases >ULN<\/strong><\/td>93 (69%)<\/td><\/tr>
LDH Increases >ULN<\/strong><\/td>78 (58%)<\/td><\/tr>
CPK Increases<\/strong> >ULN<\/strong><\/td>64 (48%)<\/td><\/tr>
Factor VIII Activity Levels >ULN<\/strong>a<\/sup><\/td>38 (29%)<\/td><\/tr>
GGT Increases >ULN <\/strong><\/td>25 (19%)<\/td><\/tr>
Bilirubin Increases >ULN <\/strong><\/td>20 (15%)<\/td><\/tr><\/tbody><\/table>
ALT=alanine aminotransferase; AST=aspartate aminotransferase; CPK=creatine phosphokinase; GGT=gamma-glutamyl transferase; LDH=lactate dehydrogenase.
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*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).2<\/sup><\/figcaption><\/figure>\n\n <\/div>\n <\/div>\n<\/div>\n\n
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Routine follow-ups keep your patients on track<\/h2>\n

Monitor ALT elevations weekly for at least 26 weeks following administration of ROCTAVIAN. See the Dosage and Administration, Administration section, Table 3: ALT Monitoring Post-Administration in the ROCTAVIAN Prescribing Information for complete monitoring schedule and guidance.2<\/sup><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Since the clinical trials began, there have been2<\/sup>:<\/h2>\n <\/div>\n\n
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References:<\/strong>
\n1.<\/strong> Data on file. BioMarin Pharmaceutical Inc.; 2024. 2.<\/strong> ROCTAVIAN\u00ae (valoctocogene roxaparvovec-rvox). Prescribing information. BioMarin International, Ltd.; 2023.<\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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RareConnectionsTM<\/sup> is here to help your patients from the start of treatment\n<\/h2>\n\t\t\t\t\t\t \t\t\t\t\t\t \t\t\t\t\t\t

Learn about available treatment support and education<\/p>\n\t\t\t\t\t\t \t\t\t\t\t <\/div>\n\t\t\t\t\t

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