Complete adverse effect profile including incidence rates and management
Important Safety Information
This is not a complete list of all possible side effects. Contact your healthcare provider if you experience any unexpected symptoms. For serious or life-threatening side effects, seek emergency medical attention immediately.
6 ADVERSE REACTIONS The following adverse reactions are also discussed in other sections of the label: Infusion-Related Reactions [see Warnings and Precautions (5.1) ] Hepatotoxicity [see Warnings and Precautions (5.2) ] Most common adverse reactions (incidence ≥ 5%) were nausea, fatigue, headache, infusion-related reactions, vomiting, and abdominal pain.
( 6 ) Most common laboratory abnormalities (incidence ≥ 10%) were ALT, aspartate aminotransferase (AST), lactate dehydrogenase (LDH), creatine phosphokinase (CPK), factor VIII activity levels, gamma-glutamyl transferase (GGT) and bilirubin > ULN.
( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc.
at 1-866-906-6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
The safety data described in this section reflect the exposure of 134 adult patients with severe hemophilia A (defined as residual factor VIII activity ≤ 1 IU/dL) to a single dose of 6 × 10 13 vg/kg of body weight of ROCTAVIAN.
Patients with detectable pre-existing antibodies to AAV5, active infections, history of thrombosis, immunosuppressive disorders, and liver dysfunction were excluded.
All patients had a median follow-up of 162 weeks (range: 66 to 255 weeks) [see Clinical Studies (14) ] .
The most common adverse reactions (≥ 5%) to ROCTAVIAN were nausea, fatigue, headache, infusion-related reactions, vomiting, and abdominal pain.
The most common laboratory abnormalities (≥ 10%) to ROCTAVIAN were ALT, AST, LDH, CPK, factor VIII activity levels, GGT, and bilirubin > ULN.
5 WARNINGS AND PRECAUTIONS Infusion-related reactions: Infusion reactions, including hypersensitivity reactions and anaphylaxis, have occurred.
Monitor during and for at least 3 hours after ROCTAVIAN administration.
If symptoms occur, slow or interrupt administration and give appropriate treatment.
Restart infusion at slower rate once symptoms resolve.
Discontinue infusion for anaphylaxis.
Like all medications, Roctavian can cause side effects. However, not everyone who takes this medication will experience them. Many side effects are dose-dependent and may improve as your body adjusts to the medication. Others may require dose adjustment or medical attention.
Contact your healthcare provider promptly if you experience:
Seek immediate emergency medical care if you experience signs of: